Social Participation, Self-Perception, and Social …...ii Social Participation, Self-Perception, and Social Support in Boys Living with Hemophilia Aubrey Serah Chiu Master of Science

Social Participation, Self-Perception, and Social Support in Boys Living with Hemophilia

by

Aubrey Serah Chiu

A thesis submitted in conformity with the requirements for the degree of Master of Science

Institute of Health Policy, Management, and Evaluation University of Toronto

© Copyright by Aubrey Serah Chiu, 2015

ii

Social Participation, Self-Perception, and Social Support in Boys

Living with Hemophilia

Aubrey Serah Chiu

Master of Science

Institute of Health Policy, Management, and Evaluation University of Toronto

2015

Abstract

Social integration is a critical component of quality of life and understanding how persons with

hemophilia interact socially with their peers may inform healthcare providers.

A random sample of 50 boys (under 18 years of age) with hemophilia from The Hospital of Sick

Children completed measures of social participation, self-perception, and social support. Parents

or guardians of participants completed a measure assessing the impact of chronic illness on the

family.

The majority of boys reported no significant participation restrictions. Social participation was

strongly correlated with self-perception subscales Social Acceptance and Global Self-Worth.

The Athletic Competence subscale was strongly correlated with social participation for

adolescents only. There were strong correlations between social participation and perceived

social support from parents, teachers, and classmates and moderate correlations between social

participation and support from close friends. There was minimal impact on the family and weak

correlations between social participation and impact on the family.

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An association between social participation, self-perception, and social support was

demonstrated in boys with hemophilia. Boys with hemophilia have minimal social participation

restrictions and are on par with their peers.

iv

Acknowledgments

First and foremost I would like to thank my supervisor, Dr. Brian Feldman, for his unwavering

support throughout my graduate work. I have learned so much over the years from you, not only

with regards to my education, but also about life. Thank you.

My thesis committee members have provided valuable advice and feedback. Dr. Victor

Blanchette’s years of service in the hemophilia field greatly influenced my husband’s life and

inspired this work. Dr. Maru Barrera’s advice and guidance informed the development of this

project. I am honored to have worked with you both.

I would like to thank the participants in both Canada and Brazil for their time and effort to

complete this study.

I also thank Audrey Abad, Marjorie McLimont, Pam Hilliard, and the rest of the research staff of

the Feldman lab for their support and guidance throughout this process. Working with you has

been a pleasure and I greatly appreciate your efforts.

This research was supported by funding from Baxter Healthcare Corporation.

Finally, I would like to thank my husband, puppy, family, and friends for their love and support,

and for giving me the opportunity to pursue my passion.

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Table of Contents

Acknowledgments .......................................................................................................................... iv

Table of Contents ............................................................................................................................ v

List of Tables .................................................................................................................................. x

List of Figures ................................................................................................................................ xi

List of Appendices ........................................................................................................................ xii

Chapter 1 Introduction .................................................................................................................... 1

1 Introduction ................................................................................................................................ 1

Chapter 2 Background and Review of Literature ........................................................................... 4

2 Background and Review of Literature ....................................................................................... 4

2.1 Introduction ......................................................................................................................... 4

2.2 Hemophilia .......................................................................................................................... 4

2.2.1 Summary ................................................................................................................. 8

2.3 Participation ........................................................................................................................ 9

2.3.1 Quality of Life ......................................................................................................... 9

2.3.1.1 Studies with Children ............................................................................. 11

2.3.1.2 Studies with Adults ................................................................................. 12

2.3.1.3 Studies with Children and Adults ........................................................... 14

2.3.2 Summary ............................................................................................................... 15

2.3.3 International Classification of Functioning, Disability, and Health (ICF) ........... 15

2.3.4 Social Participation ............................................................................................... 18




2.3.5 Summary ............................................................................................................... 27

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2.4 Psychosocial Issues in Hemophilia ................................................................................... 27

2.4.1 Depression ............................................................................................................. 28

2.4.2 Self-Perception ...................................................................................................... 30


2.4.2.2 Studies with Children and Parents .......................................................... 34

2.4.3 Summary ............................................................................................................... 36

2.4.4 Social Support ....................................................................................................... 37




2.4.5 Summary ............................................................................................................... 41

2.5 Impact on the Family ........................................................................................................ 42

2.5.1 Studies with Parents .............................................................................................. 43

2.5.2 Older Studies ......................................................................................................... 47

2.5.3 Summary ............................................................................................................... 49

2.6 Literature Critique ............................................................................................................. 49

2.7 Summary ........................................................................................................................... 51

Chapter 3 Methods ........................................................................................................................ 53

3 Methods .................................................................................................................................... 53

3.1 Research Aims .................................................................................................................. 53

3.2 Research Question ............................................................................................................ 53

3.3 Rationale ........................................................................................................................... 53

3.4 Significance ....................................................................................................................... 53

3.5 Hypotheses ........................................................................................................................ 54

3.6 Study Design ..................................................................................................................... 55

3.6.1 Ethical Issues ........................................................................................................ 56

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3.6.2 Participants ............................................................................................................ 57

3.6.3 Sample Size ........................................................................................................... 59

3.6.4 Recruitment ........................................................................................................... 60

3.6.5 Administration ...................................................................................................... 60

3.7 Data Collection Instruments ............................................................................................. 61

3.7.1 Demographics ....................................................................................................... 61

3.7.2 Health Information ................................................................................................ 62


3.7.4 Self-Perception ...................................................................................................... 64

3.7.5 Social Support ....................................................................................................... 66

3.7.6 Impact on the Family ............................................................................................ 67

3.8 Data Management ............................................................................................................. 67

3.9 Analysis ............................................................................................................................. 68

3.9.1 Participation Scale ................................................................................................ 68

3.9.2 Missing Data ......................................................................................................... 68

3.9.2.1 Single Imputation ................................................................................... 68

3.9.2.2 Sensitivity Analysis ................................................................................ 69

3.9.3 Demographics ....................................................................................................... 69

3.9.4 Correlation Coefficients ........................................................................................ 70

3.9.5 Independent Samples t-tests .................................................................................. 70

3.9.5.1 Model Checking ..................................................................................... 70

3.9.6 Multiple Linear Regressions ................................................................................. 71

Chapter 4 Results .......................................................................................................................... 73

4 Summary of Results ................................................................................................................. 73

4.1 Introduction ....................................................................................................................... 73

4.2 Enrolment .......................................................................................................................... 73

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4.3 Participant Characteristics ................................................................................................ 73

4.4 Family Situation ................................................................................................................ 74

4.5 Treatment .......................................................................................................................... 74

4.6 Internal Consistency .......................................................................................................... 75

4.7 Outcomes .......................................................................................................................... 75


4.7.2 Self-Perception ...................................................................................................... 77

4.7.2.1 Children .................................................................................................. 81

4.7.2.2 Adolescents ............................................................................................. 84

4.7.3 Social Support ....................................................................................................... 86

4.7.4 Family Impact ....................................................................................................... 91

4.8 Model Checking ................................................................................................................ 91

4.8.1 Normality of Residuals ......................................................................................... 91

4.8.2 Influential Outliers ................................................................................................ 92

4.8.3 Straight-Line Relationship and Homoscedasticity ................................................ 92

4.8.4 Non-Parametric Tests ............................................................................................ 92

4.9 Sensitivity Analysis .......................................................................................................... 92

4.9.1 Worst-Case Scenario ............................................................................................. 93

4.9.2 Best-Case Scenario ............................................................................................... 93

4.10 Linear Modeling ................................................................................................................ 93

4.10.1 Self-Perception ...................................................................................................... 93

4.10.1.1 Model Checking ..................................................................................... 94

4.10.1.1.1 Normality of Residuals ................................................................................ 94

4.10.1.1.2 Influential Outliers ....................................................................................... 94

4.10.1.1.3 Straight-Line Relationship and Homoscedasticity ...................................... 94

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4.10.2 Social Support ....................................................................................................... 95

4.10.2.1 Model Checking ..................................................................................... 95

4.10.2.1.1 Normality of Residuals ................................................................................ 95

4.10.2.1.2 Influential Outliers ....................................................................................... 95

4.10.2.1.3 Straight-Line Relationship and Homoscedasticity ...................................... 96

4.10.3 Stratification Variables ......................................................................................... 96

Chapter 5 Discussion .................................................................................................................... 97

5 Interpretation of Findings ......................................................................................................... 97

5.1 Sample ............................................................................................................................... 97

5.2 Social Participation ........................................................................................................... 99

5.3 Self-Perception ................................................................................................................ 102

5.3.1 Primary Hypothesis ............................................................................................. 104

5.4 Social Support ................................................................................................................. 106

5.4.1 Secondary Hypothesis ......................................................................................... 108

5.5 Impact on the Family ...................................................................................................... 109

5.5.1 Tertiary Hypothesis ............................................................................................. 111

5.6 Exploratory Analyses ...................................................................................................... 111

5.6.1 Self-Perception .................................................................................................... 111

5.6.2 Social Support ..................................................................................................... 111

5.7 Study Limitations ............................................................................................................ 112

5.8 Future Directions ............................................................................................................ 114

5.9 Conclusions ..................................................................................................................... 115

References ................................................................................................................................... 117

Appendices .................................................................................................................................. 124

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List of Tables

Table 1. Inclusion and Exclusion Criteria ..................................................................................... 59

Table 2. Sample Size ..................................................................................................................... 60

Table 3. Data Collection Instruments ........................................................................................... 61

Table 4. Participation Scale for Kids Questions (Open-Ended) ................................................... 64

Table 5. Participant Characteristics (N = 50) ................................................................................ 74

Table 6. Self-Perception Subscale Scores for Children & Adolescents (N = 50) ......................... 77

Table 7. Comparing the Means of the Self-Perception Subscale Scores for Children &

Adolescents (N = 50) .................................................................................................................... 81

Table 8. Self-Perception Subscale Scores for Children (n = 30) .................................................. 81

Table 9. Self-Perception Subscale Scores for Adolescents (n = 20) ............................................. 85

Table 10. Social Support Subscale Scores for Children & Adolescents (N = 50) ........................ 86

Table 11. Comparing the Means of the Social Support Subscale Scores for Children &

Adolescents (N = 50) .................................................................................................................... 86

Table 12. PedsQL™ Family Impact Module Results and Correlation with the Participation Scale

for Kids (N = 50) ........................................................................................................................... 91

Table 13. Parameter Estimates for Linear Regression of Social Participation and Self-Perception

....................................................................................................................................................... 94

Table 14. Parameter Estimates for Linear Regression of Social Participation and Social Support

....................................................................................................................................................... 95

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List of Figures

Figure 1. The International Classification of Functioning, Disability, and Health Framework .. 16

Figure 2. Study Design ................................................................................................................ 56

Figure 3. Social Participation and Social Acceptance (Children & Adolescents) (N = 50) ........ 78

Figure 4. Social Participation and Global Self-Worth (Children & Adolescents) (N = 50) ........ 79

Figure 5. Social Participation and Athletic Competence (Children & Adolescents) (N = 50) .... 80

Figure 6. Social Participation and Social Acceptance (Children) (n = 30) .................................. 82

Figure 7. Social Participation and Global Self-Worth (Children) (n = 30) ................................. 83

Figure 8. Social Participation and Athletic Competence (Children) (n = 30) ............................. 84

Figure 9. Social Participation and Athletic Competence (Adolescent) (n = 20) ......................... 86

Figure 10. Social Participation and Parental Support/Regard (N = 50) ....................................... 88

Figure 11. Social Participation and Teacher Support/Regard (N = 50). ....................................... 88

Figure 12. Social Participation and Classmate Support/Regard (N = 50) .................................... 89

Figure 13. Social Participation and Close Friend Support/Regard (N = 50) ............................... 90

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List of Appendices

Appendix A: Research Ethics Board Approval Letters .............................................................. 125

Appendix B: Data Collection Instruments .................................................................................. 129

Appendix C: Family Situation .................................................................................................... 141

Appendix D: Treatment .............................................................................................................. 141

Appendix E: Internal Consistency .............................................................................................. 142

Appendix F: Item Responses for Social Participation Scale for Kids ........................................ 143

Appendix G: Reference Scores from the Manual for the Self-Perception Profile for Children

(Harter, 1985a) ............................................................................................................................ 145

Appendix H: Social Support Scale for Children Scores by Age Group ..................................... 145

Appendix I: Reference Scores from the Manual for the Social Support Scale for Children (Harter,

1985b) ......................................................................................................................................... 145

Appendix J: Comparison with Reference Scores ........................................................................ 146

Appendix K: Normality of Residuals ......................................................................................... 146

Appendix L: Residual Plots ........................................................................................................ 147

Appendix M: Non-Parametric Tests ........................................................................................... 155

Appendix N: Sensitivity Analysis ............................................................................................... 157

Appendix O: Model Checking for Self-Perception .................................................................... 158

Appendix P: Model Checking for Social Support ...................................................................... 159

Appendix Q: Linear Modeling .................................................................................................... 160

1

Chapter 1 Introduction

1 Introduction Hemophilia is a genetic bleeding disorder caused by coagulation factor deficiency that leads to

impaired clotting in males. There are two types of hemophilia: A (lack of coagulation factor

VIII) and B (lack of coagulation factor IX). Severity of hemophilia ranges from mild (5%-40%

of normal factor levels), moderate (1%-5%), to severe (less than 1%) (Carcao, 2012; Plug et al.,

2008).

Patients with more serious disease experience recurrent bleeding into joints which can lead to

severe joint damage (hemophilic arthropathy), and impairment in quality of life (Gringeri & Von

Mackensen, 2008). Adults with severe hemophilia who have not received the benefit of long-

term prophylaxis often have restricted physical health, resulting from years of bleeding into

joints.

Hemophilia is a rare disease, which may impact the individual’s quality of life. Treatment

advances in the last 50 years have greatly improved the management of hemophilia and

increased life expectancy (Carcao, 2012; Gringeri et al., 2004), such that a primary goal for

hemophilia patients in developed countries is achieving the highest quality of life possible

(Coppola et al., 2011).

Quality of life is a difficult concept to define and there is no universal agreement on its

definition. When the World Health Organization set out to measure quality of life in 1997, they

defined quality of life as an “individuals’ perception of their position in life in the context of the

culture and value systems in which they live and in relation to their goals, expectations,

standards and concerns. It is a broad ranging concept affected in a complex way by the person’s

physical health, psychological state, level of independence, social relationships, personal beliefs

and their relationship to salient features of their environment” (World Health Organization,

1997). In contrast, health-related quality of life has been defined as “the subjective perception of

the impact of health status, including disease and treatment, on physical, psychological, and

social functioning and well-being” (Leidy, 1999, as cited in Szende et al., 2003).

2

The International Classification of Functioning, Disability, and Health (ICF) is a framework

developed by the World Health Organization that attempts to describe the multiple components

of health (World Health Organization, 2001). This framework looks at both the functioning and

disability of the individual, as well as the contextual factors that may have a direct or indirect

impact on social participation. The inclusive nature of the ICF, and its defined constructs,

provides a good framework for considering aspects of one’s quality of life.

Social participation in patients with hemophilia has been defined differently in different studies.

Some authors have defined social participation by engagement in sport (Fromme et al., 2007;

Heijnen, Mauser-Bunschoten, & Roosendaal, 2000; Köiter, Van Genderen, Brons, & Nijhuis-van

der Sanden, 2009), while others have measured participation by involvement in full-time work

(Plug et al., 2008; Van Genderen, van Meeteren, Fischer, & Helders, 2012). Oftentimes,

participation consists of measuring whether or not the individual engages in a specific activity.

Such a definition of social participation does not account for one’s desires or interest in that

specific activity. Lack of engagement in sport may be a result of inability to participate due to

physical limitations (e.g. inability to run due to arthropathy), a lack of desire to participate (e.g.

dislikes sports, prefers videogames), or lack of opportunity (e.g. parents prohibit child from

engaging in activity). Much like the construct of quality of life, the varying definitions of social

participation make discussions about social participation difficult.

Self-perception is a personal factor that may be associated with social participation. Difficulties

with emotional and psychological well-being are often reported for patients with hemophilia,

including higher numbers of depressive symptoms, lower self-perception, and lower self-esteem

than their peers (Coppola et al., 2011). Self-esteem has often been viewed as different in boys

with hemophilia compared to their healthy peers (Thomas & Gaslin, 2001). Self-perception may

influence social participation, as individuals with lower self-perception may not feel confident

enough to engage in activities of social participation. Alternatively, it may be that social

participation influences self-perception, as those who engage in social participation less often

than their peers may feel isolated and have lower self-perception.

Social participation may also be associated with social support, an environmental factor. Social

support may come from one’s family, classmates, other patients with hemophilia, from the

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patient’s healthcare team, and others, which may help to reduce social isolation (Khair, Holland,

& Carrington, 2011). With adequate social support patients may be enabled to actively

participate in their own healthcare management (Petrini & Seuser, 2009). Patients may also have

increased social participation, as they will have an increased network of people with whom to

engage in activities. Again, the inverse may also be true as patients who participate socially may

interact with others and gain more social support.

The impact of hemophilia on the family may be associated with social participation as well.

Impact on the family may be physical, psychological, or financial (Coppola et al., 2011) and can

cause significant strain on the family. How the family is able to cope with the stressors

associated with hemophilia may impact their support of social participation. For example, if a

parent perceives that engaging in sport will cause strain to the family (e.g. more frequent injuries,

the need to infuse factor more often, etc.), the parent may be less likely to encourage their child

to participate in sport. On the other hand, participating in social activities may ease some of the

psychological burden of living with illness and may lessen the impact of the disease on the

family.

The rationale for this study is to understand which personal factors relate to, and may be

associated with, social participation. No studies have directly measured the association between

self-perception, social support, impact on the family, and social participation to date in boys with

hemophilia. Hemophilia is a disease with a high burden of illness, and efforts to improve health-

related quality of life may be assisted by understanding how patients interact socially with their

peers.

4

Chapter 2 Background and Review of Literature

2 Background and Review of Literature

2.1 Introduction

A literature review was conducted using PubMed.gov using the search terms hemophilia, quality

of life, health-related quality of life, social participation, sport, participation, social support, and

impact on the family. A snowball literature search ensued as relevant articles cited in papers

initially identified were located and reviewed. Relevant articles cited in these papers were then

reviewed as well until saturation. Reviewed articles were categorized by studies with children,

with adults, and with both children and adults.

2.2 Hemophilia

Normal hemostasis requires intact blood vessels, a coagulation system that functions normally,

and an adequate number of functioning platelets (Blanchette, Sparling, & Turner, 1991).

Hemophilia is caused by deficiencies of coagulation factor VIII (hemophilia A) or coagulation

factor IX (hemophilia B) (Blanchette et al., 1991; Carcao, 2012), resulting in a defective intrinsic

pathway leading to a severe bleeding disorder (Blanchette et al., 1991).

Hemophilia is a recessive X-linked bleeding disorder that primarily affects males and equally

impacts all ethnic groups (Carcao, 2012; Feldman et al., 2008; Franchini, Frattini, Crestani, &

Bonfanti, 2012; Giannelli, 1989). Hemophilia in females is possible, but extremely rare

(Giannelli, 1989). Although hemophilia is an inherited bleeding disorder, approximately one

third of newly diagnosed hemophilia cases have no family history of the disorder (Blanchette et

al., 1991).

The existence of the two factor deficiencies was not clearly demonstrated until 1952 (Giannelli,

1989), and traditionally hemophilia A and B have been considered to have essentially identical

clinical symptoms and signs (Blanchette et al., 1991; Carcao, 2012; van Dijk, Fischer, van der

Bom, Grobbee, & van den Berg, 2005). Current prevalence rates suggest that hemophilia A

occurs in approximately 1 in 5,000 males and is the most common of all the severe inherited

5

bleeding disorders, while hemophilia B is more rare and occurs in approximately 1 in 30,000

males (Carcao, 2012; Franchini et al., 2012; Tuddenham, 1989).

A recent study by Tagariello et al. (2009) suggests that the two factor deficiencies may not be as

similar as once thought. A retrospective study with Italian patients treated between 1987 and

2007 determined that the proportion of patients undergoing joint arthroplasties was higher for

patients with hemophilia A than hemophilia B. The strengths of this study were that it included

information on patients from all the hemophilia treatment centers in Italy that conducted joint

arthroplasty surgery (268 patients from 29 centers). Due to the retrospective nature of the study,

a weakness was that detailed histories of the patients were unavailable and may not have

included relevant information such as bleeding frequency and pain. Another weakness in this

study was that a greater proportion of hemophilia B patients were living with human

immunodeficiency virus (HIV) co-infection (47% versus 26%) and thus increased mortality in

the hemophilia B population may have led to fewer patients requiring arthroplasty. The authors

did address this concern and conducted a survival time analysis to determine if other factors such

as viral infections and inhibitor status affected the odds and after adjusting for these factors, the

risk of arthroplasty for hemophilia A patients was still three-fold higher than hemophilia B

patients. Thus, patients with hemophilia B may have a less severe bleeding phenotype and better

long-term outcomes than patients with hemophilia A (Tagariello et al., 2009).

Hemophilia is typically classified into three levels of severity based on either clinical bleeding

symptoms or plasma procoagulant levels. However, due to the risk of confusion when using

clinical bleeding symptoms as criteria, it is recommended that plasma procoagulant levels be

used for classification of severity (G. C. White et al., 2001). Severity of hemophilia is not evenly

distributed amongst the population, with approximately 50% of patients having the severe

phenotype (Fromme et al., 2007). When measuring the concentration of factor that the

individual has at baseline, hemophilia is classified as follows: severe (less than 0.01 IU/ml),

moderate (0.01-0.05 IU/ml), and mild (0.05-0.40 IU/ml) (Franchini et al., 2012).

Patients with the most severe phenotype may bleed spontaneously and repeatedly without signs

of any obvious trauma (Carcao, 2012) and typically present with clinically significant bleeding,

often within the first year of life (Blanchette et al., 1991). Minor trauma to a patient with

moderate hemophilia may result in prolonged bleeding responses, while patients with mild

6

hemophilia are likely to have abnormal bleeding only in response to major trauma such as

injuries, tooth extraction, or surgery (Franchini et al., 2012). Trauma refers to physical injury

and for boys with hemophilia, trauma may include seemingly innocuous events such as being hit

by a soccer ball during physical education class. Clinically significant bleeding may include

prolonged bleeding following venipuncture or surgical procedures, but also excessive bruising

(Blanchette et al., 1991). Patients with less severe forms of hemophilia may not present with

abnormal bleeding until they are much older (Blanchette et al., 1991). Severity of hemophilia

will be reflected in the manifestation of clinical symptoms.

One of the clinical hallmarks of hemophilia is repeated joint bleeding. Recurrent bleeding into

the joints may lead to the development of joint disease and arthropathy, resulting in chronic pain

and disability (Carcao, 2012; Franchini et al., 2012). Acute joint hemorrhages may involve joint

swelling, warmth of the skin around the joint, pain, and range of motion loss (Blanchette et al.,

1991). Approximately 75% of all bleeding events in hemophilia are joint bleeds (hemarthroses)

and the most commonly afflicted joints are known as index joints (ankles, knees, and elbows)

(Carcao, 2012). A target joint is a joint that suffers from repeated bleeds, broadly defined by

some clinicians as three bleeds in three to six months (Carcao, 2012). Hemophilic arthropathy

may eventually develop in target joints resulting in synovial hyperplasia, destruction of the

articular cartilage, loss of joint space, subchondral bone cysts, bony damage, and atrophy of

surrounding bones and muscles (Carcao, 2012; Kern, Blanchette, Stain, Einarson, & Feldman,

2004). Over time, the joint may become so severely deformed and dysfunctional that the

individual is crippled by reduced mobility (Carcao, 2012). Joint bleeding is a serious concern for

patients with hemophilia.

Physical restrictions may impact social participation by limiting the activities that the patient can

engage in. Even without severe hemophilic arthropathy, chronic pain is commonly present in

patients and increased pain sensitivity is related to the severity of the joint pathology (Hilberg,

Czepa, Freialdenhoven, & Boettger, 2011). The effects of chronic pain and reduced mobility on

social participation could range from occasionally missing a social activity to complete isolation

from an activity and/or one’s peers.

Treatment for hemophilia patients is principally based on preventing or controlling bleeding

episodes with rapid and effective clotting factor replacement by intravenous infusion (Blanchette

7

et al., 1991; Franchini et al., 2012; Thomas & Gaslin, 2001). The amount of factor needed and

the timing of therapy depends on the minimal factor level required to stop or prevent bleeding as

well as the half-life of the transfused factor under ideal conditions (Blanchette et al., 1991).

Great strides in treatment advances have resulted in safe and highly effective recombinant factor

VIII and IX clotting factor concentrates, however they are extremely expensive (Feldman et al.,

2008). A study of insurance claims made to commercial health plans estimated that in 2004

dollars, the mean annualized total cost of care was USD$90,942 for patients with hemophilia,

excluding patients with HIV or hepatitis C virus co-infection (Tencer, Friedman, & Li-McLeod,

2007). As this study utilized a database of commercial health plan members, it may be biased

towards a higher annualized cost as patients with health insurance may be more inclined to use

factor prophylactically, whereas an individual without health insurance may only use factor

when absolutely necessary. Furthermore, patients with hemophilia were identified by insurance

claims: if the claimant’s insurance file included at least one record of primary hemophilia

diagnosis and at least one claim for a hemophilia drug, the patient was presumed to have

hemophilia. However this may have excluded mild and moderate hemophilia patients with few

clinical symptoms. Nevertheless, the cost of hemophilia drugs was (and continues to be) very

high. Some countries, including Canada, have allocated extra financial support to hemophilia

patients to access recombinant factors so that regular infusion of factor concentrates can be

achieved (Franchini et al., 2012).

Prevention of bleeding episodes involves both physical and psychosocial support and

comprehensive care treatment centers are often well-equipped to provide such support as their

teams are comprised of physicians, nurses, social workers, psychologists, physiotherapists, and

other healthcare professionals (Thomas & Gaslin, 2001). Physical support revolves around the

promotion of prophylaxis for the treatment of hemophilia. Primary prophylaxis involves

regularly scheduled clotting factor replacement therapy before any significant joint disease has

occurred, while secondary prophylaxis starts at a later stage, after joint disease has already

occurred (Feldman et al., 2008; Young et al., 2006). The scheduling of the prophylactic dose

may be dependent on upcoming activities that the patient may engage in. For example, a patient

who has physical education class twice a week may choose to infuse on the mornings of that

class to prevent possible injury.

8

Psychological support for hemophilia may be just as crucial for both patients and their families.

A psychologist can help individuals identify the patient’s needs, hopes, and aspirations as well as

promote self-management of one’s disease (Coppola et al., 2011). It has been suggested that

routine depression screening, assessment, education, and intervention be offered to patients with

hemophilia and that the well-being of patients with hemophilia can be enhanced by promoting

social interaction, community, networking, education, and self-reliance (Iannone et al., 2012).

Comprehensive treatment centers allow the work of a psychologist to be conducted in

cooperation and integration with the medical team.

Patients with hemophilia are a particularly fascinating population because the care of hemophilia

has undergone many dramatic changes in the past fifty years, including the development of

comprehensive care hemophilia treatment centers, home care and prophylaxis, the development

of factor concentrates, discovery of genes for both factor VIII and factor IX, and the beginnings

of gene therapy (Carcao, 2012; Nathwani et al., 2011). In the last decade, the quality and

expectancy of life for hemophilia patients have reached similar levels as those of non-hemophilic

healthy males in countries where patients have access to comprehensive hemophilia management

(Carcao, 2012; Franchini et al., 2012). Unfortunately, in countries where patients do not have

access to expensive factor concentrates, a normal life span is not yet attainable (Carcao, 2012)

and the expense of factor concentrates continues to be the greatest single barrier to the

implementation of prophylaxis around the world (Feldman et al., 2008). With comprehensive

care, patients with hemophilia receive excellent medical care and are growing up to live

relatively normal lives. Despite the advances in treatment, hemophilia continues to have an

impact on the lives of patients and thus the social ramifications of hemophilia need to be further

assessed.

2.2.1 Summary

Hemophilia is a serious chronic illness that mostly affects males. For patients with mild

hemophilia, clinically significant bleeding may only happen with major trauma. For patients

with moderate to severe hemophilia, bleeding may occur after minor trauma or may be

spontaneous.

9

Recurrent bleeding into the patient’s joints may eventually lead to the development of joint

disease and arthropathy, which can result in chronic pain and disability. This can lead to

physical restrictions, which may impact the patient’s activities and their social participation.

2.3 Participation

Social integration is a critical component of quality of life. Participation in clubs or cultural and

religious groups might facilitate social development as it provides the opportunity for children to

create friendships, strengthens the relationship between the individual and their family, exposes

them to other worldviews, and enables them to develop skills such as cooperation (McGee,

Williams, Howden-Chapman, Martin, & Kawachi, 2006). Participation in extracurricular

activities has been shown to be associated with higher levels of social self-concept and perceived

strengths (Rosenberg, 1965; Williams & McGee, 1991, and Marsh, 1992, as cited in McGee et

al., 2006).

2.3.1 Quality of Life

Only fifty years ago, management of hemophilia was focused on preventing premature death and

most patients faced the reality of crippling joints due to arthritis. Great strides in hemophilia

treatment have been achieved in the last few decades, such that physicians and patients in the

developed world have been able to shift their focus towards improving quality of life (Gringeri &

Von Mackensen, 2008). Medical improvements such as comprehensive care and access to factor

concentrates come at a substantial monetary cost, thus improvements in the quality of life of

patients with hemophilia need to be accounted for when determining the impact of treatment on

patients and their families (Gringeri & Von Mackensen, 2008; Kern et al., 2004; Naraine et al.,

2002). Due to the extremely high cost of factor replacement, it is important that the non-

monetary and non-physical benefits of prophylaxis are measured. In a literature review of

studies of patients with hemophilia, Coppola et al. (2011) found that prophylaxis was associated

with higher health-related quality of life, general health, mental health, social functioning, and

physical functioning.

Despite the excellent comprehensive care available to patients today, prophylaxis does not

eliminate all bleeds and the development of target joints or hemophilic arthropathy may still

10

occur, leading to chronic pain and/or reduced mobility. The consequences of hemophilia

continue to have a substantial impact on the affected individual’s quality of life.

Although there are many instruments designed to measure quality of life, there is still no

operational definition of this concept that is universally accepted. Many studies that purport to

investigate “quality of life” do not define the construct explicitly and use quality of life

measurements that are in fact studying other aspects of a patient’s health such as “health status”

or “functional status” (Gill & Feinstein, 1994).

The World Health Organization defines quality of life as a multi-dimensional construct

concerning an “individuals’ perception of their position in life in the context of the culture and

value systems in which they live and in relation to their goals, expectations, standards, and

concerns” (The WHOQOL Group, 1995). For a person with hemophilia living in North

America, being able to engage in a popular sport such as basketball may be expected. Quality of

life for that individual can be impacted by his ability or inability to achieve that goal. Since

engaging in activities with others is standard in North America, it stands to reason that social

participation contributes to one’s quality of life.

The concept of health-related quality of life attempts to narrow the very wide scope of the quality

of life construct. Health-related quality of life looks specifically at the extent to which one’s

health status impacts one’s life. Health status can include the disease itself or its treatment,

either of which can affect the individual’s physical, psychological, and social functioning as well

as their sense of well-being (Leidy, 1999, as cited in Szende et al., 2003). For example, a person

with hemophilia may find his health-related quality of life to be significantly impacted when he

sustains a joint bleed. The joint bleed may prevent him from engaging in the sport that he

enjoys, which can impact his physical functioning (his joints will be swollen and painful),

psychological functioning (he may be frustrated or upset that he cannot play the sport), and

social functioning (he can no longer participate with his teammates). In this example, health-

related quality of life is lowered because of his inability to engage in the sport with his

teammates and thus the individual’s social participation has also been impacted.

According to Calman (1984), good quality of life may be achieved when an individual’s hopes

and aspirations are met and fulfilled by their life experiences. Thus, to improve quality of life, a

narrowing of the gap between that individual’s expectations and their life experiences is

11

necessary (Calman, 1984). For a patient with hemophilia who desires to be a star basketball

player, good quality of life may require him to find ways to engage in the sport while taking

precautions to prevent joint bleeds. This will allow him to achieve the goals that contribute to

his quality of life and minimize the impact of his disease on his health-related quality of life.

Social participation is a critical element of both quality of life and health-related quality of life.

2.3.1.1 Studies with Children

In a cross-sectional study looking at the health-related quality of life in European children with

hemophilia, overall health-related quality of life was found to be satisfactory but the domains

that challenged the patients differed with age (Gringeri et al., 2004). This study used the first

hemophilia-specific quality of life questionnaire developed, the Haemo-QOL, and was

administered via interview for the youngest children in the sample, while older children

completed a paper version. Younger children (aged 4-7 years) were found to be impaired in the

dimension of family, which the authors inferred to be related to parental overprotection.

Children aged 8-16 were impaired in the dimension of interaction with friends and perceived that

they did not receive sufficient support from others. The oldest age group of children, aged 13-

16, reported difficulty dealing with their hemophilia. Younger children were bothered by

prophylaxis, showing lower scores in the domain of feeling, while adolescents on prophylaxis

thrived more than their on-demand counterparts in the domain of sport and school. This study

was large, spanning 20 hemophilia treatment centers in six West European countries with 318

participants. Eight centers declined to participate in the study, but there was no rate of refusal

regarding the individual participants. Without detailed refusal information, it was unclear if the

study group differed from those who chose not to participate. This study clearly demonstrated

that quality of life is not static, and changes with age and with treatment regimens.

A recent study of quality of life in Shiraz, Iran, also studied children aged 8-16 with hemophilia

using the Haemo-QoL (Bagheri et al., 2012). The authors found that impairment in quality of

life was most strongly found in the domains of friends, perceived support, and family. Although

adolescents had more impairment in quality of life than children, the mean scores for overall

quality of life showed a high degree of impairment regardless of age. None of the patients in this

study were treated with prophylaxis; all patients were treated on-demand and most patients

reported being infused by nurses at the hemophilia clinic (52%) or by their parents (41%). The

12

authors theorized that the lack of prophylaxis and participation in sports might have accounted

for the low quality of life observed in their sample. The authors identified family income, age,

mothers’ education, number of referrals to the hemophilia clinic, number of bleeding events,

number of school absences, severity of disease, person who administered infusions, insight to

health status, and insight to suffering from hemophilia as predictors of quality of life. These

variables were indicated as accounting for 90% of the variance of the quality of life. The

strengths of this study were that the authors approached all eligible patients in their clinic and

included participants’ reasons for refusal (nearly 80% of approached patients participated in the

study). One weakness of this study was the small sample size, however the authors included

their sample size calculation and achieved 90% of the required patients for 80% power. This

study clearly demonstrates that quality of life is still impaired in countries where prophylactic

therapy is not readily available.

2.3.1.2 Studies with Adults

In the United Kingdom, Miners et al. (1999) demonstrated that adults with severe hemophilia

generally had poorer levels of health-related quality of life on two separate measures than

comparative peers and those with mild or moderate hemophilia. Despite the significant

differences between groups, the majority of severe hemophilia patients reported ‘no’ or only

‘some’ difficulties in the health-related quality of life domains of the EuroQol (EQ-5D). Those

who did report severe problems tended to be older in age and similar findings were found on the

Short-Form 36 Health Survey (SF-36), where physical functioning and physical role limitation

were negatively associated with increasing patient age. On both the EQ-5D and the SF-36,

patients with severe hemophilia reported lower levels of health-related quality of life than the

general UK population (Miners et al., 1999). It is important to note that none of the patients in

this study were treated with prophylactic clotting factor. This study was conducted by postal

survey and all patients registered at a clinic were sent invitations and questionnaires to

participate (67% response rate). Strengths of this study were its large number of participants (n

= 168), good response rate, the use of well-validated generic measurements, and the inclusion of

detailed information about the non-respondents. The authors found that the participants in the

study were significantly older than the non-responders and within the sample, moderate and mild

hemophilia participants were significantly older than severe hemophilia participants. This study

13

could have been enhanced if it had included information about joint damage but nonetheless

clearly shows reduced health-related quality of life outcomes for adults with hemophilia.

Naraine et al. (2002) demonstrated that for the patient with hemophilia, health-related quality of

life was largely determined by the trade-off between the negative and positive aspects of

treatment. This study utilized the standard gamble technique, which is the gold standard for

measuring utility by determining the risk of death one would be willing to trade in exchange for

a cure for a health state. For example, a participant may have been asked to imagine that they

had a hypothetical health state such as hemophilia. They would then be asked if they would be

willing to take a pill if a cure was 100% probable. The probability of a cure was then varied

incrementally until a point of indifference was reached. The goal was to determine how much

the participant was willing to gamble for the chance of cure versus the alternative of living with

the guaranteed hypothetical health condition. This study encompassed three different

populations allowing for comparisons to be made between groups: general population (n = 30),

parents of children with hemophilia as proxy respondents (n = 30), and adults with hemophilia (n

= 28). They concluded that hemophilia and its treatment resulted in a reduction in health-related

quality of life but that prophylaxis was preferred over on-demand treatment regimens. A

weakness of this study could lie in how it framed the two alternatives as this could have had a

profound effect on the subjects’ decisions. The authors were aware of this potential for bias and

strived to present the scenarios in as balanced and neutral a manner possible. The authors found

that the treatment of hemophilia and the complications of treatment had a negative impact on

health-related quality of life.

In Austria, Hartl et al. (2008) conducted a case-control study reporting significantly lower

health-related quality of life scores for adult patients with hemophilia on the SF-36 domains of

physical functioning, role-physical, bodily pain, and general health than control subjects (Hartl et

al., 2008). Mental health scores were not significantly different between the patients and the

controls. There was a good participation rate for the cases in this study as 64% of the patients

eligible from two hemophilia centers participated in the study (n = 53). There was some

difficulty obtaining controls for this study and thus 65% of the control group was comprised of

age-matched, male acquaintances of the authors from the same geographic area as the patients (n

= 104). However, given that the controls were acquaintances of the authors, it is not

unreasonable to speculate that the controls may have also differed systematically in another way

14

from the patients. Although patients and controls had similar educational backgrounds, there

may have been differences in socioeconomic status, employment, or other variables. That being

said, this study offers further evidence that physical limitations may be the primary barrier that

patients with hemophilia face.

More recently, the SF-36 was administered to patients with hemophilia in Germany in a cross-

sectional study. Similar results were found with significant differences between patients and

controls by Hilberg et al. (2011) primarily in domains of physical functioning, role-physical, and

bodily pain. Again, there were no significant differences when it came to the mental health

domain. A weakness of this study was that it had a smaller sample of just 36 patients and there

was no discussion of participation rate or reasons for non-participation. However the method of

recruiting controls included recruitment by newspaper advertisement and from the local

community. Recruiting controls in this manner may be more balanced than the aforementioned

method of recruiting via author networking, although the controls did differ from the patients in

terms of employment status in this study (a significantly higher proportion of controls were

employed compared to the patients). This study further supports the notion that physical

limitations have the most significant impact on quality of life for patients with hemophilia.

2.3.1.3 Studies with Children and Adults

A literature review of hemophilia studies including both children and adults noted that one of the

most widely used generic quality of life instruments was the SF-36 (Fischer, van der Bom, & van

den Berg, 2003). The authors concluded that patients with hemophilia have lower scores in

physical domains of health when assessed by quality of life measurements. When it came to

mental health, however, quality of life scores were often on par with normative means. Strengths

of this study were that they only examined studies that used validated instruments, eliminating

studies that may have drawn conclusions from non-validated instruments. Weaknesses of this

literature review were that it was limited to articles written in the English language and specified

medical subject headings (MESH) of ‘hemophilia A’. These search parameters may have been

too stringent and may have excluded studies in non-English speaking countries, which may have

different treatment protocols and thus different manifestations of clinical symptoms. It may also

have excluded research with hemophilia B patients. Nonetheless, this review provided insight

into the state of health-related quality of life in hemophilia as all thirteen studies reviewed

15

reported that hemophilia reduces health-related quality of life in physical health when compared

to the general population.

As evidenced by the increasing amount of research in the field of hemophilia, achieving high

quality of life is an important and relevant topic of study. However, none of the above studies

clearly defined what good quality of life meant or what the individual domains represented to

their patients. Without a clear definition of the construct, research is inherently limited and

comparison of studies is near impossible. Gill and Feinstein (1994) called on investigators to

clearly define what they meant by the term ‘quality of life’ and to identify the domains to be

measured. To answer that call, rather than attempting to study all the domains of health-related

quality of life, this research project was undertaken to critically examine just one domain of

quality of life in hemophilia: social participation.

2.3.2 Summary

Measurement of the construct ‘quality of life’ is difficult as there is no universally accepted

operational definition. However, those measures that do exist for measuring quality of life show

that overall, health-related quality of life for patients with hemophilia is satisfactory.

Despite the advances in hemophilia treatment, the consequences of the disease continue to have

an impact on patient’s quality of life. Studies have shown that patients with hemophilia tend to

have lower health-related quality of life scores in the physical domains than healthy controls, but

no significant differences are found when it comes to the mental health domains.

When quality of life in patients with hemophilia is assessed, patients with hemophilia tend to

have lower scores than their healthy peers only in the realm of physical health.

2.3.3 International Classification of Functioning, Disability, and Health (ICF)

The World Health Organization developed a framework for describing the components of health

called the International Classification of Functioning, Disability, and Health, known as the ICF

(World Health Organization, 2001). The ICF is arranged in two parts, each with two

components:

16

Part 1) Functioning and Disability

• Body functions and structures

• Activities and participation

Part 2) Contextual Factors

• Environmental factors

• Personal factors

Figure 1 illustrates the ICF model, which states that a person’s functioning and disability is the

result of the dynamic interaction between the person’s health conditions and the contextual

factors in their life (World Health Organization, 2001).

Figure 1. The International Classification of Functioning, Disability, and Health Framework

Functioning and disability begins with the physiological functions of the body systems, whereas

the body structures are the anatomical parts of the body (World Health Organization, 2001). In

the case of hemophilia, the body’s ability to clot normally is a body function that is impaired and

may result in damage to the body structures such as the individual’s joints.

17

There has been much debate over the definition of activities and participation, however the ICF

attempts to define activities as “the execution of a task or action by an individual” while

participation encompasses “involvement in a life situation” (World Health Organization, 2001).

While running may be an activity that an individual can perform, running with one’s classmates

during physical education class would be an example of participation. In other situations, the

differences between activities and participation may be more difficult to distinguish. To assist in

differentiating between activities and participation, we use the term ‘social participation’ to

reinforce the social aspects involved in a life situation. A person’s limitations or restrictions of

activities and/or participation are assessed against the norm, or the generally accepted population

standard (World Health Organization, 2001). In the case of hemophilia, studies often assess the

individual’s limitations or restrictions in comparison to an individual without hemophilia or with

some other type of chronic illness.

Contextual factors are those factors that compose the individual’s complete background,

including both environmental and personal factors (De Kleijn, Heijnen, & Van Meeteren, 2002).

The contribution of each of these factors and the dynamic interaction between them will

determine the level and the extent of that person’s functioning (World Health Organization,

2001), including their social participation.

Environmental factors include the physical, social, and attitudinal environments within which

individual’s live and conduct their lives (World Health Organization, 2001). They may be

positive or negative influences on a person’s life and they may be at the individual or societal

level. For example, a patient with hemophilia who is wheelchair-bound but who has a

wheelchair accessible home has an individual environmental factor that has a positive influence

in their life. However, societal attitudes towards wheelchair accessibility that inform laws and

regulations may have a negative impact on his life if the places he frequents are not wheelchair

accessible. Thus the environmental factors that an individual encounters can greatly shape his or

her activities and participation.

Personal factors compose “the particular background of an individual’s life and living, and

comprise features of the individual that are not part of a health condition or health states” (World

Health Organization, 2001) and may include things such as gender, race, age, self-perception,

etc. These contribute to one’s life experience, again in both positive and negative ways. The

18

male patient with hemophilia may be able to command a higher salary than his female peer, a

positive influence for him (and a negative influence on the female peer’s life). However, he may

have difficulty finding employment if he is a member of a racial minority in a non-inclusive

society. Again, the personal factors of an individual will influence his or her activities and

participation.

2.3.4 Social Participation

It has been suggested that children with chronic illness “may carry high social and emotional

price tags” (La Greca, 1990, as cited in K. A. Williams & Chapman, 2011) and for these

children, social participation restrictions may in fact be more important to them than their disease

(van Brakel et al., 2006). The inability to participate like one’s peers could both lead to and

result in stigma, which may further restrict one’s social participation (van Brakel et al., 2006).

Trying not to appear ‘different’ from one’s peers may become of primary importance to the child

with chronic illness, especially if the illness impacts the physical appearance of the child. While

some patients may find the invisibility of hemophilia a positive because the disease can be

hidden from others, some have reported struggles as a direct result of its invisibility (K. A.

Williams & Chapman, 2011). Thus the patient may go to extreme measures—and engage in

extreme activities—to prove that he is just like his peers.

For persons with hemophilia, participation in the same activities as one’s peers is not always

possible. Joint or muscle bleeds that frequently plague the patient are often followed by resting

(Heijnen et al., 2000), limiting his ability to participate. Pain, swelling in joints, decreased

mobility, restrictions in activities, worries about bleeding, and the discomfort and inconvenience

associated with treatment may all contribute to reduced sport participation or play with their

peers for the boy with hemophilia (Thomas & Gaslin, 2001; Young et al., 2006). Loss of muscle

tone could result from reduced physical activity that could further facilitate hemarthrosis,

hemorrhages, and joint damage (Buzzard, 1996). Even when younger children are allowed to

play sports, as children get older and the sports become more competitive and rough, the child

may have to stop playing due to the risk of bleeds (Beeton, Neal, Watson, & Lee, 2007). If the

patient suffers from target joint disease or hemophilic arthropathy, there may be more serious

consequences and the patient may require hospitalization, physical therapy, splints, orthotics, or

surgery, further alienating him from his peers (Kern et al., 2004).

19

Sport participation is seen to be a normal and desirable aspect of socialization in Western society

(Heijnen et al., 2000), and the advances in hemophilia treatment have made participation in

physical activities, including sports, a possibility that had previously been denied. Treatment in

developed countries makes it possible for children with hemophilia to participate in nearly all

sports (Köiter et al., 2009) and given that home treatment is now widely present for Western

European and North American children, one can expect that these patients with hemophilia will

be able to participate as much as their peers in nearly all physical activities (Carcao, 2012;

Gringeri et al., 2004). In fact, sport is not only a possibility for patients but is now widely

recommended to improve both psychological and physical well-being by healthcare

professionals, the World Federation of Hemophilia, national member organizations, and the

pharmaceutical industry (Carcao, 2012; Heijnen et al., 2000; Von Mackensen, 2007; Mulder,

Cassis, Seuser, & Narayan, 2004). The prevailing attitude is that patients with hemophilia

should participate in all school activities they can while respecting the limitations of their disease

(Heijnen et al., 2000). Thus it comes as no surprise that in the case of hemophilia, sport

participation has received a significant amount of attention in recent years.

The ability to participate in virtually any sport is a significant achievement, but does not take into

consideration other modes of participation that may be motivated by a patient’s interest. Social

participation needs an element of desirability. Studies on social participation that focus solely on

sport are only able to identify sports non-participation, as they lack the breadth needed to

describe social participation. Research must account for the fact that some people do not like

certain activities, including sport. For example, research could include measurement of social

participation such as spending time with friends, engaging in social activities, or participating in

social events. That being said, participation in sport and physical activity should still be

encouraged for all persons by stressing the benefits of a healthy lifestyle for joint protection and

hemorrhage reduction (Buzzard, 1996). When recommending activities, both physical and non-

physical, the patient’s personal preferences and interests should be taken into account.


The treatment regimen that a patient is on can influence his participation. Gringeri et al.’s (2004)

study of children and adolescents with hemophilia in Western Europe found that patients treated

with prophylaxis were less impaired when it came to sports and school than patients who

20

received on-demand treatment. Specifically, adolescents on prophylactic treatment reported less

impairment in the domains of ‘sport and school’ than those who were treated on-demand. The

authors concluded that the dimension of sport and school revealed higher health-related quality

of life. The authors assumed that patients who participated more in sport and school must have

experienced higher quality of life than those who did not. Again, this was a large study with

many participants (n = 318), but without information on non-participants and non-random

sampling methods, generalizability may have been somewhat limited. Nevertheless, one can see

that a patient’s treatment regimen can greatly impact that patient’s social participation.

In the United Kingdom, Khair, Holland, and Carrington (2011) conducted an initial focus group

with seven boys with hemophilia aged 9-17, which revealed that the patients were eager to

participate in sporting activities and knew that their treatment enabled them to do so. These

patients reported that “having hemophilia did not prevent them from being ‘normal teenagers’”

(Khair et al., 2011). The focus group was transcribed and interpretive analysis was applied, and

although the sample was small, the purpose of this focus group was to gather information about

the social networking habits and preferences of adolescents rather than to gauge social

participation. A questionnaire was developed based on the focus group discussions and less than

a third (31%) of the invited patients participated (n = 47). From this subsample, 70% of patients

endorsed the statement that they were not able to play some sports. Given that the focus of the

questionnaire study was on social networking, it is possible that patients who did not engage in

these activities chose not to participate in the study, which may explain the low response rate. It

is evident from this study that boys with hemophilia desire to engage in sporting activities but

there are still activity restrictions that they face.

Meanwhile in the Netherlands, Köiter et al. (2006) found that boys with hemophilia aged 8-18

were engaging in sport with a mean frequency of five times a week, with the most popular sports

including soccer, swimming, and tennis. This study found that older boys tended to be more

cautious when choosing sports with a higher risk than younger boys, suggesting that they

recognized the risk of bleeding associated with such sports and acted accordingly. This hospital-

based cohort study included 158 participants from five hemophilia treatment centers and had a

response rate of 65%. This study included information about non-responders and the authors

found no significant differences in the patient’s diagnosis, age, height, weight, and body mass

index between participants and non-participants. Including this information was crucial and

21

made the generalizability of this data more viable. Not all the participants completed the

questionnaires on sports and when additional analysis with non-responders was conducted, it was

revealed that the boys who completed the questionnaire on sports were significantly younger

than those who did not. It may be that only those who engaged in sport completed this

questionnaire and in line with Heijnen et al.’s (2000) results, younger patients may be

participating more in sports than older patients. Alternatively, it could be that adolescents were

less interested in completing questionnaires and may be equally involved in sports as younger

patients. From this research, one can again conclude that boys with hemophilia are engaging in

sports.

Even if children with hemophilia are able to participate at the same level as their healthy peers in

physical activities, it may or may not coincide with increased health-related quality of life

according to van der Net et al. (2006). They found that participants aged 8-14 in their cross-

sectional study engaged in physical activities at school, extracurricular sports, and leisure time

activities for 245 minutes per week, lower than the recommended norm of 420 minutes per week.

All the patients had normal functional ability and relatively high quality of life. Physical fitness

was defined by maximal exercise testing and only weak relationships were found between

physical fitness and health-related quality of life. This study only included patients with severe

hemophilia A, thus we do not have any information about children with different subtypes of

hemophilia. However this study raises the very important question of whether or not physical

activities should be used as the benchmark for high quality of life.

Recent research in Australia found that the majority of children with hemophilia did not meet the

national physical activity guidelines of at least 60 minutes of moderate to vigorous physical

activity per day (Broderick, Herbert, Latimer, & van Doorn, 2013). The authors found that only

43% of all children with hemophilia met the government guidelines for physical activity, in

contrast to healthy peers who met the guidelines 57% of the time in the winter and 67% of the

time in the summer. When active, children with hemophilia participated mostly in low or

medium risk activities, in keeping with recommendations to avoid contact sports. Children with

hemophilia aged 4-18 participating in a case-crossover study nested in a prospective cohort study

provided data on their activity using a validated, retrospective, interviewer-assisted, modifiable

activity questionnaire (Kriska’s MAQ) and prospective activity diaries. Over half of the eligible

population in the three eastern states of Australia participated (n = 104) and most of the patients

22

were receiving prophylactic treatment (86%). The strengths of this study were its large sample

size and its use of both retrospective and prospective data to form a more detailed picture of the

participants’ activity levels. Some of the weaknesses of this study were that the retrospective

activity questionnaire might have been subject to recall bias as the participants were asked to

recall physical activity in the previous 12 months while the prospective activity diaries had a

poor follow-up rate (64%). However, this study shows that even where prophylactic therapy is

available, boys with hemophilia are engaging in physical activity at a different rate than their

healthy peers.


Participating in the workforce is a different type of social participation that persons with

hemophilia may strive to achieve when they grow older. Plug et al. (2008) found that in the

Netherlands, severe and moderate hemophilia patients between the ages of 16 and 64 were able

to participate actively in the workforce. However, patients with severe hemophilia tended to

participate less in full-time paid work when compared to men in the general population. Patients

who were treated with prophylaxis tended to engage more in the workforce. Those patients who

were part of the workforce experienced restrictions in their job performance due to hemophilia.

Patients with severe hemophilia were also more likely than the general population to report

having complete occupational disability. Patients with hemophilia reported less full-time

employment, but higher participation rates in education than their peers. When comparing

patients who were employed to those who were unemployed, differences were mainly found

between physical functioning, role limitations due to physical problems, and role limitations due

to emotional problems. For patients who have moderate or mild hemophilia, differences when

compared to their peers were only slight or not at all. This study was part of a series of nation-

wide postal surveys in the Netherlands and the 2001 series received a high response rate of 68%.

Of those who responded, 68% were eligible for analyses (due to age of patient and availability of

employment information), which still amounted to a large sample size (n = 721). The authors

stated that the non-responders did not appear to differ from the responders in severity or type of

hemophilia, but were slightly younger. The representativeness of the sample was one of the

strengths of the study, but without information about the respondents who completed data for the

Central Bureau of Statistics, it was unclear if the general population these patients were

compared to was representative of the Dutch male population. That being said, the likelihood of

23

representativeness is high and this study allows us to conclude that although daily activities for

the person with hemophilia may be limited due to joint problems or viral infections, hemophilia

has “only a minimal influence on social participation” for adults (Plug et al., 2008).

For patients with hemophilia and viral infections, participation in the workforce can be

particularly hampered. In the same study by Plug et al. (2008), 25% of patients who were

infected with hepatitis C virus (HCV) and 7% of patients infected with HIV were occupationally

disabled. The side effects of treatment are temporary but serious, which may have impacted

participation.

Unemployment rates of hemophilia patients vary from country to country, but in some countries

it can be particularly problematic. Although in the Netherlands it was found that the percentage

of unemployed adults with hemophilia was similar to that of healthy controls (Plug, 2004),

patients with hemophilia in Austria were four time as likely to be unemployed compared to

controls (Hartl et al., 2008). As discussed earlier, the study by Hartl et al. (2008) used controls

that were acquaintances of the authors, and thus may have skewed the results in a negative

fashion. In the United States, one study found that 46% of patients were unemployed (Iannone et

al., 2012).

Marriage is another form of social participation that adult patients may wish to engage in. In

Hartl et al.’s (2008) study of Austrian patients with hemophilia, a significantly higher number of

patients compared to healthy controls were married. Individuals in the control group were more

likely to have partners with whom they were not married. There were no differences between

patients and controls when it came to individuals without a partner. This was in contrast to an

earlier study published in 1990 by Rosendaal et al., which found the exact opposite: the

percentage of patients with hemophilia from the Netherlands who were married was significantly

lower than the general population (13% fewer). This sample found that 44% of the patients

considered their disease to be an impediment when it came to finding a partner and 27% of the

patients found that their disease had a negative effect on an existing relationship. This study

involved a postal survey mailed to all registered patients with hemophilia in the Netherlands and

used national data to compare patients to the general population (Rosendaal et al., 1990). A

large proportion of invited patients responded (over 80%, n = 935) and the mean age of the

sample was only slightly younger than the general population. With such a high response rate, it

24

is easier to generalize these findings to the hemophilia population than the aforementioned

Austrian study; however 18 years elapsed between the two studies. In that time, there were

significant changes in disease treatment and morbidity, which may account for some of the

differences in marital status. That being said, neither of these studies incorporated questions

about desire for marriage and Hartl et al. (2008) found that a large number of controls had

partners to whom they were not married. Even though hemophilia may have been seen as an

impediment to finding a partner, this study did not speak to patients who were in relationships

who may not wish to be married.

A recent study from the Netherlands examined coping in adults with severe hemophilia and

briefly looked at participation (Binnema, Schrijvers, Bos, Schuurmans, & Fischer, 2014). The

Impact on Participation and Autonomy Questionnaire was used to measure participation, where

higher scores indicated more restrictions in participation. The majority of patients (58%)

reported few participation restrictions with scores ≤ 30 on a scale with a maximum score of 120.

Strengths of this study were the large number (n = 86) and the wide age range (aged 18-68) of

participants. One of the weaknesses of the study was that 19 patients were excluded as they had

retired or were unemployed and therefore could not complete the participation questionnaire.

The excluded patients tended to be older and differed in health status from those who

participated, so including them in the analysis may have lead to very different results. However,

the inclusion of participation in this study demonstrates the importance of this area when

studying disease-related stress.


Although participating in sport may lead to negative consequences for boys with hemophilia,

Irish respondents in an observational, cross-sectional study by Sherlock et al. (2009) reported

social exclusion and/or participation restriction as negative experiences as well. In this sample,

60% of the patients engaged in sports and half of these patients participated in sports that have

been associated with bleeding complications. The majority of these patients did not take factor

prior to participating in the sport, either. When asked about negative experiences with sports,

61% of the respondents noted negative experiences with sports (e.g. bleeds, pain, joint damage),

13% listed social exclusion and/or participation restriction. Patients who did not engage in sport

listed the primary reasons in descending order as arthritis, pain, time constraints, and having no

25

interest. Although nearly half (48%) the respondents were able to achieve high levels of physical

activity, over a quarter (26%) of the respondents failed to reach recommended levels of physical

activity. Severity of disease was related to physical activity, with all but one patient with mild or

moderate disease engaging in moderate to high levels of physical activity. Furthermore, all

patients under the age of 30 achieved moderate to high levels of physical activity. A major

strength of this study is that the authors captured reasons for non-participation in sport and a

wide age range of patients were included in the study, from ages 16-69. However, weaknesses

of the study also include that broad age range, as patients over the age of 50 will likely have very

different sport participation than 16-year olds. The recruitment process also revealed a weakness

as 90% of the sample was a convenience sample and only 10% of the sample were randomly

selected from the national database and recruited via postal distribution. Of those who were

invited via post, there was only a 12% response rate. No information about the patients who

refused was documented. It is possible that patients who attended the hospital differed in a

systematic way from patients who did not attend, for example they may have more severe

disease or more disease complications. Although this study only represents 12% of the Irish

hemophilia population, it serves to highlight the importance to patients of being able to

participate socially as one’s peers, despite illness, pain, and possible disability.

The Netherlands has produced a number of studies focusing on hemophilia and sport

participation. Heijnen et al. (2000) conducted a study with Dutch children and adults with

hemophilia aged 6-29. The authors found in their cross-sectional study that 95% of boys below

12 years of age participated in physical education classes at school. Of those who participated in

physical education, 11% were unable to participate in all physical education activities. As

children aged, the percentage of children who were unable to participate in all physical education

activities increased to 34%. The authors found that approximately 30% of the younger boys

never missed physical education because of bleedings, however in the older patients over 60% of

them sometimes did not attend physical education due to bleeds. It was also evident from the

older sample that physical limitations increased as age increased. Of the patients aged 18-29

years, 50% were never or only sometimes able to run, while the same was true for 89% of

patients over the age of 29. It is important to note that rates of prophylaxis and intensity have

changed over the years and was likely associated with the differences found between older and

younger children’s participation in physical education classes as well as their physical

26

limitations. In general, the majority of patients. even those with severe hemophilia, were able to

participate in some kind of sport. Strengths of the study include the large number of participants,

with 293 clinic patients recruited from a wide range of ages. Importantly, the authors asked

participants who attended school whether they liked physical education class and only 2% of

patients indicated that they did not. This would indicate that participating in physical education

class like their peers was significant for the majority of respondents. Although this study was

large, it only represented approximately 23% of the Dutch hemophilia population. This study

does not include information about patients who declined to participate, limiting its

generalizability. Nonetheless, for this sample one can conclude that the hemophilia population is

physically active and that children with hemophilia are able to participate in physical education

on par with their peers.

Lindvall et al.’s (2006) study of adolescents and adults in Sweden, Denmark, and Norway found

that 86% of hemophilia patients reported having the same opportunities as their healthy peers

when it came to social activities. The authors inquired about certain social activities including

having a girlfriend, having sex, partying, consuming alcohol, sport, work, and career. Over 100

patients (n = 108) completed the cross-sectional questionnaire between the ages of 13 and 25

years with a response rate of 80%, but there was no information about the patients who declined

to participate. As this study aimed to determine treatment compliance and understanding of

disease, it is possible that only those patients with detailed knowledge and good compliance with

treatment participated. Increased treatment compliance may also provide for increased ability to

engage in social activities, possibly overestimating the social participation of hemophilia

patients.

Although one cannot conclusively say if patients with hemophilia are as engaged in the

workforce as healthy controls based on the mixed results from multiple studies, it can be

concluded that the majority of patients with hemophilia are able to participate in the workforce.

Social participation can encompass many activities in one’s life. Some of these activities will be

desirable to an individual, and others may not be. Given that participation is defined by

“involvement in a life situation” (World Health Organization, 2001), it is crucial that any study

that professes to measure social participation as a health-related quality of life outcome further

measures the individual’s desire to participate in that life situation.

27

2.3.5 Summary

It is important for patients to feel that they can participation socially as their peers. Social

participation may be impacted by hemophilia as joint or muscle bleeds could lead to pain and

discomfort, decreased mobility, and activity restriction. This may prevent a boy with hemophilia

from engaging in sport participation or playing with their peers.

Engaging in sport has been shown to improve both psychological and physical well-being.

There has been much research into sport participation, which clearly demonstrates that boys with

hemophilia wish to engage in sporting activities but that they may not be able to participate on

par with their peers.

However, physical activities are not the only markers for quality of life and research has also

shown that patients with hemophilia may be impacted in their employment, marriage, and other

social activities.

2.4 Psychosocial Issues in Hemophilia

Patients with hemophilia and many other chronic illnesses are often believed to be at higher risk

for psychosocial issues than their peers. The child with hemophilia is at risk for developing

psychosocial issues as a result of their perception of their disease, but also because of the

interpretations of others about their hemophilia (Thomas & Gaslin, 2001). It has been previously

suggested that up to 30% of all children with chronic disease may develop social and

psychological maladjustment (Pless & Roghmann, 1971, as cited in Klein & Nimorwicz, 1982).

Children with chronic illnesses like hemophilia may have difficulty feeling a sense of

empowerment and capability. Thomas and Gaslin (2001) wrote that pain, disability, and

treatment regimens might force the patient to be dependent on others rather than promoting

independence as the child progresses through developmental stages. This constant dependency

may lead to the child questioning his self-worth or capacity, further negated by well-meaning

friends and family who may try to help the patient. The authors theorized that children with

chronic illnesses may have more difficulty “mastering the developmental stages needed to

progress in a healthy and productive way” (Thomas & Gaslin, 2001).

28

The school age years are a time when children begin to explore independence from their parents,

but younger boys with hemophilia will be reliant on their parents to administer treatment. Spirito

et al. (1991) argue that dependence on one’s parents could lead to impaired peer interactions and

social adjustment later in life. Multiple clinic visits and periodic hospitalization could reinforce

dependent roles for boys with hemophilia where they must “relinquish control and subsequently

question their senses of self-worth” (Thomas & Gaslin, 2001). Finally, children may be less

socially active outside of the home than adolescents, which may lead to them reporting more

social participation restrictions.

Having access to hemophilia treatment centers and prophylactic therapy may have desirable

effects not only on the physical aspects of the disease but also the social aspects. Coppola et al.

(2011) point out that in developed countries the use of prophylaxis allows patients to live

relatively normal lives without the need for overprotection. This is a marked shift in the attitudes

towards patients with hemophilia compared to just 30 years ago and illustrates the non-clinical

benefits of prophylaxis.

2.4.1 Depression

Many have studied depression in the hemophilia population and some have noted that patients

who have negative attitudes towards having a chronic condition are susceptible to increased

levels of depression, increased behavioral problems, and lower self-esteem (Thomas & Gaslin,

2001).

A cross-sectional study of adolescents aged 15-18 with hemophilia A in Egypt by Hassan et al.

(2011) found that two thirds of the subjects reported mild to severe depression on the Beck

Depression Inventory—Short Form (BDI-SF). The authors suggested that patients with

hemophilia might suffer from depression due to aspects including poor self-image,

hemarthropathy, chronic pain, financial burden of disease, and family psychodynamics. This

study used a random sample of 50 adolescent patients who attended an outpatient clinic. This

method of recruitment ensured a representative sample of patients participated but the authors

did not disclose any information about non-responders. Nonetheless, the patients represented a

wide range of therapy regimens and the random sampling method used lends credence to the

generalizability of the study. From this study one can conclude that a large percentage of

hemophilia patients in Egypt are suffering from some level of depression, and the authors noted

29

that the rate of depression was nearly double the percentage of patients reporting to have

depression in the United States.

That study in the United States was conducted by Iannone et al. (2012) and examined the

prevalence of depression in adult males with hemophilia, 18 years or older. They found that

37% of their patients reported some level of depression on the Patient Health Questionnaire

(PHQ-9), with 20% reporting moderate to severe depressive symptoms. Although the sample is

not particularly large (n = 41), 84% of the patients enrolled at the treatment center participated in

the study suggesting representativeness of the hemophilia population in that geographical area.

Along with a questionnaire asking patients to report depressive symptoms, they were given a

depression fact sheet at annual comprehensive clinic visits that included symptoms, treatment

options, and self-care prevention. The rationale for giving this fact sheet was to counteract any

potential negative consequences of increased awareness about depression, however it may have

had the unintended consequence of sensitizing the participants to depressive symptoms, resulting

in over-reporting. All patients in the study were also given a consultation with a psychosocial

provider during the same visit, which may have led some patients to over- or under-report their

symptoms. It is clear from both of these studies that depression does impact some members of

the hemophilia population, but it is equally clear that these two studies cannot be compared as

they involve different study populations (adolescents versus adults) and different outcome

measures (BDI-SF versus PHQ-9).

More recently, Kim et al. (2013) studied depression using the Beck Depression Inventory in

adult patients with severe hemophilia in Korea and found negative associations with quality of

life as measured by the World Health Organization Quality of Life Assessment. Fifty-three

patients consented to participate, however seven were excluded due to incomplete answers

resulting in a sample of 46 patients. The strengths of this study were that it was conducted with a

homogenous sample of severe hemophilia patients with Asian ethnicity, one of the first studies to

investigate quality of life and psychological status in Asian hemophilia patients. However, one

of the limitations of this study was that the instrument used to measure depression had a

suggested cut-off score of 16 and the median of the sample was 4.0 (IQR = 0.8-11.3) (Kim et al.,

2013). Although the data may have statistical significance, the sample does not appear to

include any patients with clinically significant depression. Nonetheless, this is an important

30

study that explores quality of life and the psychological impact of hemophilia outside the

Western world.

While developed countries with adequate comprehensive care may have defined budgets for

psychosocial support, developing countries may face budgetary restrictions and scarce resources

to attend to the psychosocial needs of their patients (Coppola et al., 2011). Without adequate

therapy and social support, patients may be more at risk of developing depression and other

psychiatric disorders.

On the other hand, some studies have found that living with hemophilia does not increase the

risk of behavioral or socialization problems (Coppola et al., 2011), and some studies have shown

that living with a chronic illness may even have positive outcomes for psychological adjustment

and self-esteem (Thomas & Gaslin, 2001). Studies discussed earlier noted that when health-

related quality of life was measured using the SF-36, neither of the study samples in Austria nor

Germany suffered in the mental health domain, even when compared to controls.

2.4.2 Self-Perception

As discussed above, children with chronic illnesses such as hemophilia, may be more vulnerable

to psychosocial issues like lowered self-perception or self-esteem. Self-perception refers to

one’s perception of themselves, including their feelings of competence and adequacy in specific

domains, as well as their overall self-esteem and self-worth (Harter, 1985a). Self-perception

may be considered a type of lower-order evaluative dimension of the superordinate constructs of

self-esteem and self-worth (Harter, 1982). Marston (1990) defined self-esteem as “the degree to

which one feels lovable and capable just for existing or being. This notion of self-esteem

represents the core sense of ‘self’ and how valued the self is to the child and those individuals

who are important in the child’s life” (as cited in Thomas & Gaslin, 2001). For the patient with

hemophilia who faces hospitalization, physical therapy, or surgery, these events may result in

decreased attendance at school and may contribute to a sense of reduced capabilities and self-

esteem (Kern et al., 2004).

The patient with hemophilia may be more dependent on others, have disruptions in their social

life, and have difficulty attaining peer acceptance (Agle, 1984). Having fewer opportunities to

succeed in activities compared to one’s peers or adhering to protective measures issued by

31

parents or healthcare professionals may lower one’s self-perception, even if one is not acutely

bleeding (Hegeman et al., 2011). The disease may negatively influence self-perception as it may

disable patients from engaging in regular activities or social contacts through pain or risk of

bleeding (Hegeman et al., 2011) and it may heighten adolescent concerns of “having an

unreliable and changing body” (Agle, 1984).

The ability to self-infuse as one gets older may enable adolescents to encounter fewer

participation restrictions. Mastery of self-infusion techniques and management of one’s

hemophilia will not only enable the patient to engage in more activities, but also can contribute

to his sense of accomplishment. It also reduces his dependency on his parents and gives him a

sense of control over his illness. Indeed, there are camps dedicated to boys with hemophilia that

include the teaching of self-infusion to promote self-management. Thomas and Gaslin (2001)

described the interactive and supportive camp experience as providing opportunities for children

with hemophilia to “gain independence and ‘prove’ their self-worth and ability.” Thus, self-

infusion and self-management serve the dual purpose of promoting both social participation and

self-perception.

There is no universal conclusion when it comes to self-perception of chronically ill children

when compared to healthy peers. Despite all the potential areas where self-perception and self-

worth may be impacted by one’s disease, the literature on self-esteem often finds marked

similarity between patients and healthy controls (Kellerman, Zeltzer, Ellenberg, Dash, & Rigler,

1980). In other studies, however, multiple differences between the two groups were found.


An older study by Kellerman et al. (1980) found no differences between a group of adolescents

(aged 12 and older at a children’s hospital) with chronic illnesses and a comparison group of

healthy adolescents in a cross-sectional study in the United States on the Rosenberg Scale of

Self-Esteem. There were significant sex differences, with females endorsing lower self-esteem

than their male counterparts in both groups. The healthy peers were selected from junior high

and high school populations, while the ill group was randomly selected from various clinics at a

children’s hospital and there were no differences between the groups in terms of sex distribution,

religion, history of previous illness, or number of siblings. The ill group was significantly older

than the healthy peers in one disease group as it included patients beyond the age of high school,

32

but the authors determined through statistical analyses that any age confound was minimal. The

strengths of this study included its size, as with over 500 participants (n = 517) this study was

very large, and it represented six different disease groups (oncology, cardiology, diabetes, cystic

fibrosis, nephrology, and rheumatology). However, given the differences in ages between the ill

and the control group, one cannot help but wonder if the lack of differences may have been a

function of age. The authors did not list the age ranges of the two groups, which is a significant

omission as adolescents may be more vulnerable to lowered self-esteem. They did include the

mean age of each disease group, which is not significantly different between each disease group.

Furthermore, the measure used to assess self-esteem was a generic measure intended for an adult

population that may not have been sensitive enough to pick up differences between the

chronically ill adolescent participants and the healthy peers. This wide-sweeping study did

suggest that lowered self-esteem may not necessarily be the case for the majority of patients with

chronic illness and that overall, adolescents with chronic illness have normal levels of self-

esteem.

When it comes to being bullied and the personality traits of patients with hemophilia, differences

have been observed when compared to healthy peers. A cross-sectional study in Finland by

Kvist, Kvist, and Rajantie (1990) identified patients with hemophilia by attendance at a hospital

clinic in 1986, and of the 16 families contacted, 13 patients consented to participate. The authors

estimated that this represented 21% of the school-aged children with hemophilia in Finland. The

control group was comprised of healthy male classmates who preceded and succeeded the target

patient alphabetically (2:1 ratio). The teachers rated the boys with hemophilia to more likely be

the victims of bullying, identifying 31% of the patients with hemophilia to be bullied.

Significant differences were also found between the boys with hemophilia and the controls when

they self-rated their personality traits, with patients rating themselves as being less caring, quick,

open, and joyful but more self-assured and independent. The authors attributed the positive

effects of being given responsibility in the household to more self-assurance and independence in

the patients. The authors found no differences in school achievement or absences between the

two groups. The authors had access to data from a previous study conducted in 1971 with a

similar recruitment procedure involving 10 children with bleeding disorders and healthy

classmates at a 2:1 ratio. In the 1971 data, the achievements of the ill group never reached the

achievements of the control group. The strengths of the study were that they interviewed

33

patients, healthy controls, and teachers, plus it had the added benefit of the earlier comparison

data. However, with such a small sample size that represented less than a quarter of the school-

aged hemophilia population, the generalizability of this sample is questionable. That being said,

it appears that at the time this research was conducted in the late 1980s, there were significant

differences between patients with hemophilia and their healthy peers.

More recent data from developed countries, where patients have access to comprehensive care,

indicates that patients with hemophilia may report depressive symptoms and lower self-

perception, but not necessarily at a clinically significant level (Coppola et al., 2011). Perceived

competence may refer to one’s global self-worth, an overall reflection of the value of oneself, or

it may refer to evaluations of one’s competence in specific domains (Hegeman et al., 2011).

Trzepacz et al. (2003) found that boys aged 8-15 years in the United States with hemophilia

reported difficulties with emotional well-being, more depressive symptoms, and lower self-

perception than their peers in a cross-sectional study. When comparing the dimensions on the

Self-Perception Profile for Children, differences were found on the dimensions of Scholastic

Competence, Athletic Competence, and Global Self-Worth, however the scores were still within

the normal range. The authors found no differences in social functioning between the two

groups, as others equally identified children with hemophilia as popular, prosocial, disruptive, or

sensitive. Although children with hemophilia had higher numbers of depressive symptoms, they

were still lower than the normative mean score. Thus, even though children with hemophilia

endorsed more depressive symptoms than their peers, their scores still remained well below

clinical levels. The authors identified 50 patients who were enrolled at major medical centers

eligible for recruitment, of which 47 parents gave permission to the authors to contact their

children’s school. Eight schools declined to participate. Comparison peers were identified as the

classmate with the same race, gender, and closest birthdate to the target child, of whom 80% of

the first-choice comparison families consented. The strengths of this study were that it tested a

large proportion of children with hemophilia from the selected medical centers and had a robust

method of identifying comparison peers. Weaknesses identified in the study included the lack of

information about non-participants (both the patients and the schools) as well as the small sample

size. Furthermore, by identifying eligible patients only through comprehensive care rosters, the

authors have excluded children with hemophilia who do not receive comprehensive care (if they

exist) and who may be more vulnerable to psychosocial issues. Nevertheless, this study is an

34

excellent example of a study of how patients with hemophilia differ from their peers and the

authors concluded that they are strikingly similar. Even where differences were observed

between the patients and their peers regarding depression and self-perception, they were still

below clinically significant levels.

Patients with hemophilia in the Netherlands also encountered some difficulties with perceived

competence. Hegeman et al. (2011) conducted a cross-sectional study in five treatment centers

with patients aged 8-18 and noted that having positive self-worth and feelings of adequacy or

competence in specific domains may enable a person to better cope with daily challenges and are

key to good psychological health. They found that patients with hemophilia had self-perception

scores that were generally comparable to their healthy peers. The only significant differences

between the two groups were found around competence in Close Friendship, where the children

with hemophilia had significantly lower self-perception. This was a substudy of a larger study

with over 200 patients (n = 243) with a (65%) response rate. Only 52% of participants in the

larger study completed the main outcome questionnaires in this substudy (n = 126). Non-

responders were significantly younger than the participants but did not differ in terms of

diagnoses. The authors used reference scores provided by the Dutch version of the Self-

Perception Profile for Children and the Self-Perception Profile for Adolescents to compare the

patients with hemophilia to healthy controls. This study had a large number of participants,

although the response rate was considerably lower than some of the other studies on participation

and that may call into question the generalizability of the results. More information about the

reference group’s demographics and how they compared to the children with hemophilia would

have allowed for better comparison between the two groups. That being said, it is evident from

this study that patients with hemophilia tend to have normal levels of self-perception in many of

the domains.

2.4.2.2 Studies with Children and Parents

Emiliani et al. (2011) conducted semi-structured interviews with Italian parents of boys with

hemophilia and noted that several features of hemophilia can affect the psychological

development of patients, including the unpredictability, duration, and severity of bleeds. For

both the patient and the caregiver, this leads to a “persistent state of uncertainty” which may be

accompanied by anxiety that will further influence the developmental trajectory of the patient

35

and his family. Living with such uncertainty may make it difficult for the patient to participate

in social activities (e.g. planning soccer games).

Like other chronic illnesses, hemophilia can be a socially stigmatizing condition because patients

often face negative social consequences when they cannot participate in activities (K. A.

Williams & Chapman, 2011). Williams and Chapman (2011) recently conducted nine qualitative

interviews in the United States with boys living with hemophilia and their parents. The authors

found three main themes in the interviews: awareness of difference between the patient and his

peers, efforts by the patient and family to conceal the difference, and efforts to connect with

peers and friends. Although many of the children emphasized they were as normal as their peers,

most parents noted the differences between their child and his peers. Even though the children

reported feeling just like their peers, they had a tendency to conceal their health condition or

were very selective as to whom they disclosed their illness to. Patients were the subjects of

stigma by others as one patient reported that using crutches following joint bleeds resulted in

ridicule by his peers, as there were no obvious signs of injury. One parent described the social

isolation his son with hemophilia felt by simply stating: “He can’t participate in a lot of things

these boys do” (K. A. Williams & Chapman, 2011). Participants were identified through a

hemophilia treatment clinic and through purposive sampling with the intent of having a

heterogeneous sample. By interviewing both the parents and the children, this study was able to

more broadly capture the experiences of the patients. The children tended to downplay the

impact of hemophilia on their lives when asked directly, whereas the parental perspective

highlighted some of the differences. Strengths of the study include its detailed description of the

methods used to identify the sample, the study approach (grounded theory), and the data analysis

methods (interviews were transcribed and independently coded using open coding). Since the

purpose of the study was to understand the social challenges that the children face, the small

sample is adequate as the sampling method allowed for maximum variation. Weaknesses of the

study include the lack of discussion about how inconsistencies were resolved between the

independent coders. It is clear that hemophilia can impact participation for boys with

hemophilia, even though parents are more likely to voice the difference.

In a literature review by Von Mackensen et al. (2007), it was determined that quality of life was

influenced by the clinical aspects of one’s hemophilia as well as psychosocial variables.

Participation in social activities has many positive effects if the activities are seen by the

36

participants to be enjoyable and compatible with their peers (Heijnen et al., 2000). Researchers

have theorized that participation in sports activities has many non-physical benefits for the

patient with hemophilia, including an increased feeling of social acceptance, better social

adaptation, participating with friends and peers, providing companionship, not feeling alone,

preventing withdrawal or depression, controlling stress, increasing body image, feeling a sense

of achievement, feeling like one is living a normal way of life, increasing confidence, promoting

an independent lifestyle, and increased self-esteem (Coppola et al., 2011; Heijnen et al., 2000;

Köiter et al., 2009; Von Mackensen, 2007; Petrini & Seuser, 2009).

Parents of patients with hemophilia noted that the ability to compete in sports with their peers

increased their children’s self-esteem (Beeton et al., 2007), suggesting that the effects of

participation in sports are visible to others as well. Von Mackensen et al. (2007) concluded that

engaging in sport activities could have an impact on these psychological variables and that sports

should be advocated for persons with hemophilia. Providing patients and parents with practical

and psychological support to engage in sport activities may encourage social participation and

lead to the development of these benefits.

2.4.3 Summary

Self-perception is the way that one perceives his or herself and may include feelings of

competency, adequacy, and overall self-esteem and self-worth.

Patients with hemophilia have often been thought to be at higher risk for psychosocial difficulties

than healthy controls. Indeed, past research has shown that there were significant differences

between patients with hemophilia and their healthy peers when it came to bullying. However,

more recent research in countries where patients have access to comprehensive care has shown

that although patients with hemophilia may report more depressive symptoms and lower self-

perception than their peers, they may not reach clinically significant levels.

One can see that living with a chronic illness does not necessarily mean that one will develop

psychological difficulties. It is also clear that psychological problems are not limited to

childhood, as even patients who live for many years with their disease may incur psychological

problems later in life and could benefit from psychological support (Coppola et al., 2011).

37

Assessing the psychological needs of a patient should be an on-going process throughout an

individual’s lifetime rather than a one-time assessment.

2.4.4 Social Support

To actively engage in life’s activities, one must have the support of others. Social support

provides many clinical benefits as well as psychological benefits and may be an important factor

in social participation. Without the opportunity to engage in social activities with peers or social

isolation resulting in part from one’s disease, a patient’s self-perception and social participation

may suffer.

For patients with hemophilia, the illness may result in restricted mobility for periods of time if

the patient experiences a joint bleed. This may lead to social participation difficulties if activities

are subsequently restricted. Given that this is a time for children to develop mastery, it is

imperative that the adults that form the patient’s social support network encourage appropriate

social interaction, academic learning, and physical learning experiences (Manning, 1988).

Without the opportunity to develop independence and autonomy, there may be social difficulties

for the patient (Spirito, DeLawyer, & Stark, 1990). This may require parents/guardians and

teachers to take a step back and allow children to engage in activities even when their instinct is

to protect and shield the child from potential failure.

With comprehensive care, prophylaxis, and home treatment, the educational opportunities for

boys with hemophilia have changed as well. While historically patients with hemophilia may

have been home schooled or had multiple school absences (B. Kvist, Kvist, & Rajantie, 1990),

patients are now able to attend normal schools with fewer prolonged absences (Coppola et al.,

2011). Importantly, by integrating with normal schools, boys with hemophilia are in contact

with their peers and have greater access to social support and more social participation

opportunities.

Overprotectiveness of parents may prevent patients from participating in activities that they

would like to engage in. Traditionally, patients with hemophilia have been denied participation

in sports or any activities that could be perceived as risky (Buzzard, 1996). This was not entirely

due to parental wishes as up until the end of the 1960s, sport participation was strictly forbidden

for persons with hemophilia (Buzzard, 1996; Fromme et al., 2007; Hegeman et al., 2011; Köiter

38

et al., 2009; Von Mackensen, 2007). Although overprotective parents may forbid children with

chronic illnesses from participating in sport, it has been argued that forbidding a child to engage

in a certain activity may result in the child doing it without the parents’ knowledge (Buzzard,

1996). Furthermore, Schlieper (1985, as cited in K. A. Williams & Chapman, 2011) found that

patients restricted from participating in physical or athletic activities with their peers had

difficulties developing social skills, self-esteem, and friendships.

Parental overprotection could potentially lead to passive-dependent, daredevil, and other

negative behaviors (Agle, 1984; Beeton et al., 2007). It may also contribute to lower self-

perception as the child may feel that the parent does not believe that he is capable of making

decisions or executing tasks (Hegeman et al., 2011; B. Kvist et al., 1990). Lindvall et al.’s

(2006) sample of Scandinavian patients with hemophilia found that 33% of patients felt they

would be treated differently by their parents had they not been born with hemophilia.

Unsurprisingly, the patients believed their parents to be overprotective and anxious. The social

participation of the patient may be consequently impacted by the family’s attitudes towards

certain activities and may lead to a reduction in social support. Despite all these conjectures,

there is little evidence suggesting that parents of boys with hemophilia are overprotective (Klein

& Nimorwicz, 1982).

Some organizations have attempted to foster the development of self-esteem and encourage

social support by providing children with hemophilia with camping opportunities that give

patients a chance to gain independence, meet other boys with hemophilia, and develop self-worth

and ability (Thomas & Gaslin, 2001). In the United Kingdom, a restricted access online social

network has been launched for patients with hemophilia designed to promote disease-

management skills for adolescents (Khair et al., 2011).


Research in social support and hemophilia has found a significant relationship between social

support and health-related quality of life. In the previously discussed research study about the

health-related quality of life of patients with hemophilia in Western Europe, Gringeri et al.

(2004) found that adolescents reported impairment in the domain of perceived support.

Teenagers with hemophilia (aged 13-16 years) who suffered three or more major bleeding events

in the previous twelve months felt significantly less supported than teenagers who suffered less

39

than three major bleeding events. No difference was found in perceived support for the patients

11-13 years old when it came to the number of major bleeding events. In this sample, it appears

that as the boys get older, social support from peers becomes a more salient problem and can

impact the patient’s quality of life.

Opportunities to engage with other hemophilia patients have decreased over the years, which

may result in lowered social support. It appears that comprehensive home care has reduced the

amount of time that patients spend in hospital; therefore patients have fewer opportunities to

interact with other patients with hemophilia. Not only does this reduce peer support, but it also

eliminates the potential for patients to learn from their older peers about how to manage their

illness. In a cross-sectional study by Khair et al. (2011) adolescents (aged 10-18 years) in the

United Kingdom found that nearly half their sample (49%) had no contact with other people

outside their family who had hemophilia. However, 72% of the patients endorsed that they

would like to meet with others who have hemophilia. Respondents reported that some of the

worst aspects of living with hemophilia were that people looked at them differently (21%) and

that they felt alone (19%). This survey was distributed to 150 adolescents in 11 centers in the

United Kingdom, but only 47 questionnaires were returned (31%), compromising the

generalizability of these results. That being said, it is clear from this study that without access to

other patients with hemophilia, patients may grow up lacking peer support from someone who

understands their illness, and this may contribute to feelings of isolation.

Petrini and Seuser (2009) conducted a literature review on hemophilia care in adolescents (aged

12-18 years) and concluded that social support was the single most important factor that

influenced compliance. Studies have shown that compliance with prophylaxis tends to decline in

adolescence (Geraghty et al. as cited in Petrini & Seuser, 2009). The authors suggested that

education, encouragement, positive feedback, and active participation in the patient’s care by the

patient himself could support the perception that prophylactic therapy is part of normal life. This

can then restore or maintain compliance with prophylaxis. The patient’s support network is

ideally comprised of parents, classmates or peers, healthcare providers, and other caregivers.

Compliance with prophylaxis may enable a patient to engage in physical activities more safely

and frequently. Unfortunately, the authors of this literature review did not provide evidence as to

how they came about this conclusion. It is possible that social support may influence

40

compliance, and if so, prophylactic treatment has been shown to be associated with better social

participation.


In the study by Iannone et al. (2012) with adult hemophilia patients in the United States, it was

found that lack of social support was significantly associated with patients reporting higher

depressive symptoms. Nearly a quarter (22%) of patients reported that they lacked social

support from family, friends, or community. Patients who did not feel supported by their social

network were five times more likely to report depressive symptoms than patients who felt

supported. This study had a high response rate (84%), but a weakness of this study could be

increased sensitization to the problem of depression as the study provided patients with

information about depressive symptoms prior to administering the questionnaire. Furthermore,

given that this was a cross-sectional study, it is impossible to discern the causal direction—was it

the lack of a social network that caused the depressive symptoms or the inverse? That being

said, from this study one can conclude that perceived lack of social support may be a risk factor

that ought to be addressed by hemophilia treatment center programming.


Similar to findings by Khair et al. (2011) in the United Kingdom, Lindvall et al. (2006) found

that severe hemophilia patients 13-25 years of age in Scandinavia reported contact with other

patients with hemophilia 52% of the time, while moderate patients knew of others with

hemophilia 50% of the time. Unlike the United Kingdom sample, however, of the Scandinavian

severe patients who did not have any contact with others with hemophilia, only 27% reported

that they would like to have contact. This study had a large number of participants (n = 108) but

the participation rate was not reported. This study supports the results in the United Kingdom

and it seems apparent that today’s patients with hemophilia have limited contact with others

living with hemophilia. Patients may have limited support in this area, but they may not

necessarily desire more support from the hemophilia community.

Having close friends to disclose one’s illness may be important for patients with hemophilia. A

qualitative study with hemophilia patients in the United States suggested that close friendships

contributed to patient’s self-acceptance and self-management of their disease (K. A. Williams &

41

Chapman, 2011). In a study in Scandinavia, the vast majority (94%) of patients reported having

disclosed their illness to their friends (Lindvall et al., 2006). However, for some patients

disclosing their hemophilia to others may be a threatening or scary thought. Choosing to

withhold their disease status may be a way of protecting themselves, as some research has shown

that patients with hemophilia may be more vulnerable to bullying by their classmates (B. Kvist et

al., 1990; K. A. Williams & Chapman, 2011). Having close friends provides a “buffer against

teasing and aggression from the larger peer group,” thus disclosing to close friends as opposed to

all of one’s classmates is more beneficial and less risky (K. A. Williams & Chapman, 2011). In

a sample of adolescents in the United Kingdom, approximately half of the patients had concerns

about who was aware of their disease and one patient reported not telling anyone about their

illness (Khair et al., 2011). However, without disclosing one’s disease, it may be difficult to

develop meaningful relationships with others. This may further hamper opportunities to grow

one’s social support network and have access to social participation.

Finally, another important source of social support comes from the parents or guardians of the

patients. According to Williams et al. (2011), parents can facilitate efforts to form social

networks and bonds for their children. If parents are more supportive of a patient’s desire to

engage in a sporting activity, it will be far easier for the patient to participate in both the sporting

activity and the social aspects of the activity.

2.4.5 Summary

Patients with hemophilia have historically been home schooled or had multiple school absences,

but with the advent of newer treatments, patients are now integrated with normal schools. This

has had the added benefit of ensuring that boys with hemophilia are in contact with their peers.

However, now that treatment is based in the home (in developed countries), patients may have

fewer opportunities to engage with other hemophilia patients. Some patients may not know of

any others who have the same disease as them and this can lead to feelings of isolation. That

being said, although the patients may have limited support, they may not desire more support

from the hemophilia community either.

Patients with hemophilia have reported feeling a lack of social support and this has been

associated with increased symptoms of depression. Feeling a sense of social support may also be

42

impacted by one’s hemophilia as another study has shown that patients who have fewer major

bleeding events tend to feel more supported than patients who have more bleeding events.

Social support can come from many sources and for patients with hemophilia, it may be critically

important that he has someone to whom he can disclose his illness and other important matters.

2.5 Impact on the Family

Chronic illness does not affect the patient alone; rather it impacts the entire family. The stress

and anxiety of having a child with chronic illness can have a substantial effect on family

dynamics (Beeton et al., 2007). Furthermore, since hemophilia is a hereditary disorder

transmitted through the X chromosome, parents may feel guilty for being carriers of the disease

(DeKoven et al., 2014). If family members perceive the disease to have a high impact on the

family, they may be less inclined to encourage social participation. This may be especially

salient if such participation could lead to further joint bleeding or injury that may have additional

impact on the family. For example, a family may not be supportive of a child’s desire to play

football due to fear that the patient will be injured and require additional care (e.g. extra

infusions of factor or a trip to the hemophilia treatment center). Thus, it is imperative that there

is an understanding about how hemophilia impacts the family.

Following the initial diagnosis of hemophilia, a significant number of stressors present

themselves to the family, including the need to prepare for bleeds, emergencies, treatment, and

financial pressures (Coppola et al., 2011). Since many patients with hemophilia in North

America and Western Europe are now treated in the home, parents must play a dual role of both

parent and health care provider. How the family deals with stress and distressing emotions such

as sadness, anger, fear, and guilt, will influence the patient’s ability to adapt to his disease (Agle,

1975). Without adequate coping behaviors for adaptation to the sometimes overwhelming stress,

psychological disorders and arrests in development may occur (Agle, 1984). Throughout the

patient’s lifespan, hemophilia will act as a chronic, recurrent stressor on both the patient and his

family.

The frequent transitions between crisis and non-crisis may add further strain to the patient and

the family (Rolland, 2012). Hemophilia and its accompanying stressors can have a significant

impact on the attitudes of the patient’s support network (Coppola et al., 2011). In the literature,

43

there are reports of negative effects on the marital relationship, parent/child relationships, family

mobility, finances, and the number of children borne (Klein & Nimorwicz, 1982). This

continued strain on the family could significantly impact the patient’s social support network,

self-perception, and social participation.

2.5.1 Studies with Parents

Saviolo-Negrin et al. (1999) studied parents of children with hemophilia in Italy in relation to

coping. Almost half the families had no history of hemophilia and were thus unprepared for the

diagnosis. The authors found a high correlation between the Beck Depression Inventory (BDI,

which measures tendency to depression) and the Questionnaire of Resources and Stress (QRS,

which measures parent and family problems, pessimism, and child characteristics). The authors

also found a correlation between the “unconscious” anxiety scale and the QRS dimensions,

indicating that the mothers were unaware of their anxiety regarding the child’s health. Fathers

were found to be less involved than mothers when it came to the problems of coping but they

still experienced pessimism, anxiety, and depression. Correlations also showed that the more

stress the fathers experienced, the less they consciously accepted and had a sense of reality about

their child’s disease. Interestingly, 95% of the parents considered the child’s illness to be not

‘serious’, even though 65% of the sample had co-infections (HCV or HIV). This may have been

a function of the parents not accepting the reality of the disease or it may have reflected the

parents coping strategy (Saviolo-Negrin et al., 1999). Strengths of this study were that they

included both the mothers and fathers of the children with hemophilia and they included reasons

for refusal. Weaknesses of this study were that it was a small sample (20 couples of parents and

two mothers) but the data gained from both parents provides a more complete picture of parental

coping in Italy.

In the United Kingdom, Beeton et al. (2007) interviewed 12 parents (including three couples) of

children between the ages of 18 months and 13 years with severe hemophilia A or B to explore

the impact of hemophilia on parents. Four separate focus groups were held over a two-year

period and three individual interviews were conducted. The authors used a ‘code and retrieve’

method to label sentences and phrases according to their meaning and then collated the data to

reveal four themes: experiences, managing the condition, engaging with others, and mastery.

For some parents, the initial diagnosis caused grief, guilt, and loss of self-esteem, regardless if

44

the mother knew she was a carrier or not. Due to the uncertainty caused by unpredictable bleeds

and lack of experience with hemophilia, the most stressful period for some of the parents were

the child’s very early years. Parents also had difficulty when their children wished to play

sports, as they had to choose which sports were suitable and not suitable for the child to

participate in. It was especially difficult for parents when they had to stop their older children

from continuing to play a sport they were good at and enjoyed. Some parents felt they had to

restrict their children from the sport as the play had become rougher and more competitive with

age.

Beeton et al. (2007) also found that parental self-esteem was lowered as some parents avoided

interacting with others, further isolating themselves and emphasizing their loss of self. The

introduction of prophylaxis provided parents with an increased sense of protection for their child,

and enabled the patient to engage in more vigorous activities. However, for some parents the

added responsibility of prophylaxis was restricting and overwhelming as they felt they always

needed to be available in case of an emergency or crisis. This need for availability also imposed

restrictions on employment, because although several parents’ employers stated that the parent

could leave work in case of emergency, some parents reported that in practice, this was often

difficult and that frequent absences had a negative impact on employment.

The strengths of the study by Beeton et al. (2007) include the wide age range of the children of

the respondents, as the authors were able to probe the different challenges parents faced

throughout childhood. Another strength was the detail with which the authors described their

sample and their methods of transcription. All interviews were audiotaped, transcribed, and sent

to the parents for verification, amendments, or additional comments. Furthermore, by offering

the option of engaging in a focus group or an individual interview, the authors allowed those

participants who preferred the anonymity of an individual interview to retain such

confidentiality. There may also have been some social desirability bias in the focus groups, as

parents may not have wanted to portray their family as struggling too much with the illness

compared to their peers. Nevertheless, this study offered insight into the triumphs and

challenges parents of boys with hemophilia face and the impact of the family.

Elsewhere in the United Kingdom, Shaw and Riley (2008) conducted qualitative interviews with

parents of children between ages 8 and 12 with bleeding disorders (hemophilia and von

45

Willebrand Disease). Similar to Beeton et al.’s (2007) results, the authors found that parents

struggled with the desire for their children to live a normal active lifestyle and the desire to

protect the child from bleeding. The advent of prophylaxis allowed the patients and families

more freedom to navigate the world. Prophylaxis was less disruptive to the family life and

reduced absences from school and work for the patient and his family; it had a normalizing effect

for parents. Parents perceived the negative impacts of prophylaxis to include struggles with

injections, especially when the child was young or if the child was resistant to treatment and did

not cooperate with injections. While home-based treatment meant that parents were no longer at

the hospital for extended periods of time, the parents noted that they missed opportunities to

interact with other parents of children with hemophilia. Parents desired social support from

other parents with children living with chronic illnesses. These families were identified through

two comprehensive care centers and of the 27 families invited to take part, 24 families consented

to participate. Strengths of the study included the heterogeneity of the sample as the families

represented two types of diseases and a wide range of treatment. However the authors did not

detail their methods of gathering data or how the data was analyzed. There was also no

discussion of the ethical procedures or the authors’ experiences in qualitative works.

Nonetheless, these qualitative studies in the United Kingdom demonstrate clearly that

hemophilia affects not only the patient, but also the family in profound ways.

Semi-structured individual interviews were conducted in Italy with families of children with

hemophilia to learn about the process of normalization (Emiliani, Bertocchi, Potì, & Palareti,

2011). Normalization involves communication and relationships with other family members to

understand the disease and establish new, more adaptive habits. Their study revealed that

families who coped well with the illness had learned to live with hemophilia as they searched for

normality within their lives. They also adapted to daily life with hemophilia by integrating

hemophilia treatment within the family’s daily routine whenever possible. By making

hemophilia a part of their lives rather than maintaining a divided concept of life between the

normal and the sick, families were better able to cope with the daily pressures of life. This may

further allow the families to encourage the patients to engage in normal activities of life,

including social participation with one’s peers.

Detailed case studies of two families affected by hemophilia are also described by Emiliani et al.

(2011) to support their findings. One family had normalized hemophilia, a large social network,

46

and found prophylaxis to be “more a bother than an ordeal.” In contrast, the other family was

dominated by the illness and its manifestations, closed to other social supports, and the mother

felt direct responsibility for the child’s illness.

In the first phase of this study, all families with children under the age of majority were invited

to participate and 10 of the 16 families contacted agreed to participate, representing young

children and teenagers with hemophilia. Five years later, the three participating families who

had since started prophylaxis were invited to participate in another interview (the remaining

families were still using on-demand therapy). All interviews were audiotaped and transcribed

then analyzed using thematic analysis of elementary context to identify shared themes or issues.

The strengths of this study were the case studies, which eloquently describe the normalization

process. The follow-up interviews with families who switched to prophylaxis also added richness

to the study. Other strengths were the detailed descriptions of the analysis methods, ethics

approval obtainment, and gathering of informed consent. The in-depth account of the families

and their different modes of adaptation offer valuable insight into the impact of the disease on

families.

Breakey et al. (2012) administered to 44 parents of patients with hemophilia the Pediatric

Quality of Life (PedsQL™) Family Impact Module (FIM), which had previously never been

applied to the hemophilia population. The authors found that there was a moderately strong

positive correlation between the FIM and the quality of life for parents of children with

hemophilia. As only the abstract has been published thus far (Breakey et al., 2012), it is unclear

how the parents scored on the FIM but this study does suggest that parental quality of life can be

impacted by the child’s chronic illness.

In the United States, DeKoven et al. (2014) found that caregivers of children with hemophilia

identified multiple domains as burdensome, most notably the domains child’s pain, emotional

stress, financial, transportation, sacrifice, and medical management. In the domain of child’s

pain, most caregivers reported ‘nearly always’ being burdened when their child complained of

joint and bleeding pain and wished they could take the pain away. Interestingly, although

children were not feeling much pain at the time of the survey, caregivers reported a very high

burden when they saw their children in pain. Using a general linear model, the authors identified

current presence of an inhibitor, lower family income, higher number of bleeding episodes in the

47

past six months requiring a trip to the hospital/hemophilia treatment center, and greater lost

productivity to be significantly associated with caregiver burden. The authors conducted an

online cross-sectional questionnaire using a mailing list of hemophilia caregivers and patients.

Although 681 caregivers were invited to participate, 339 subjects initiated the survey, and the

final sample included 310 caregivers (46% response rate). One of the strengths of this study was

that it included detailed information about the response rate, but a weakness of this study was

that it was administered to a convenience sample from an opt-in research mailing list with an

honorarium of $100. The honorarium may have biased the sample towards caregivers with

lower income brackets and may not have been representative of all hemophilia caregivers. That

being said, this recent study has demonstrated that there is a significant burden on the caregivers

of hemophilia patients.

2.5.2 Older Studies

Earlier research into the impact on the family tends to suggest that hemophilia had a larger

impact on the family before the advent of comprehensive care and prophylaxis. Interviews

conducted with children with hemophilia and their parents in the United States found that many

of the patients described hospitalization at some time and noted that bleeding episodes prevented

them from participating in activities (Browne, Mally, & Kane, 1960). They found that children

with hemophilia appeared to be outwardly docile and passive, but would rebel in subtle ways.

Rorschach tests with the patients revealed underlying active and aggressive urges. They

concluded that a major psychological problem for the patients was their conflict about activity,

as wishes for rapid movement lead to anxiety. Mothers who were carriers of the disease felt

intensely guilty for having passed the disease to their children. The authors described the

mothers as “depressed, anxious women who tearfully discussed their hemophilic children,” many

of whom tried to account for the child’s bleeding in non-genetic ways (e.g. diet and trauma

during pregnancy lead to the hemophilia). Mothers were least anxious when their child was at an

age where he could be confined to a crib or playpen; as the child grew older and began to walk,

play with other children, and go to school, the mothers’ anxiety levels increased. Most fathers

were anxious and relieved that they had not contributed to the patient’s disease genetically; some

were described as aloof and remote. Most of the fathers felt that their child was capable of doing

more than he was allowed and felt that the mothers were overprotecting the child.

48

The families in Browne, Mally, and Kane’s (1960) study were selected based on a population of

49 patients already undergoing clinical and laboratory studies, of whom 40 parents agreed to

participate, representing a range of children from 1-16 years of age. One of the strengths of this

study was the heterogeneity of the sample, however the authors did not provide any framework

to their interview or detail their methods of analysis. The authors included three case studies to

highlight the impact of disease at all levels of severity, and it was clear that even mild disease

could have a significant impact on the family. The authors went so far as to conclude that

hemophilia was a disease of both the mother and the child.

Varekamp et al. (1990) studied the impact of hemophilia on the mothers of patients in the

Netherlands. They found that when it came to daily practical problems, most mothers responded

that they almost never felt their activities or relations were disturbed because of extra care

needed for the patient (77%-96%, depending on the type of activity). Mothers said that the

following activities were disturbed sometimes: the family’s daily activities (20%), housekeeping

(19%), relaxation after work (19%), going out with their partner (17%), going on holidays with

the family (16%), and time for hobbies (15%). When it came to marital relations, more than half

of the married respondents (52%) said that their child’s hemophilia had no impact on their

relationship, and 45% indicated that they had grown closer together. However, 44% of the

respondents who were divorced felt that their child’s illness had contributed to the divorce.

Mothers of severe hemophilia patients tended to have higher levels of psychological distress than

mothers of mild or moderate patients. There was also a negative correlation (-0.3) between

maternal feelings of guilt and perceived support from the partner; the authors suggested that this

might validate the hypothesis that maternal guilt leads to overprotective behavior of the mother

and disengagement of the father.

There was a very high participation rate in Varekamp et al.’s (1990) study with 110 mothers

consenting of the 126 invited to participate in the study (87%). One of the weaknesses of this

study is that although they described the differences between disease phenotype and

psychological distress, they did not indicate if the levels of psychological distress found were

clinically significant. The authors did compare their scores to that of another study with mothers

of children with epilepsy and noted that the epileptic mothers had higher levels of distress than

the hemophilia mothers, suggesting that there is less psychological distress in the hemophilia

parental population. This quantitative study of the burden of disease in the late 1980s illustrates

49

what living with hemophilia was like nearly thirty years ago after the development of modern

hemophilia treatment (but before recombinant technologies) and during the HIV/AIDS (acquired

immunodeficiency syndrome) epidemic.

2.5.3 Summary

The above studies make it clear that chronic illness is something that does not affect the

individual, but rather it impacts the family as a whole. The initial diagnosis of hemophilia may

be followed by feelings of grief, guilt, and low self-esteem for the parents. Just like patients

need social support, parents may desire social support from other parents of children with

chronic illness to reduce feelings of isolation. Again, since treatment in developed countries now

takes place in the home, there may be fewer chances for parents to interact and reach out for

support.

Even with modern hemophilia treatment and comprehensive care, parental emotional well-being

is as vulnerable to the setbacks of hemophilia as the patients. While there has been much talk

about patient-centered care, family-centered care may be more applicable to the patient with

hemophilia and his family. Some parents have expressed a desire for professional support during

these difficulties (Beeton et al., 2007). By identifying families in crisis or in need of further

support to make decisions on appropriate levels of activity for the patient, one might be able to

enhance the patients’ social support, self-perception, and social participation.

2.6 Literature Critique

There has been much research on health-related quality of life in patients with hemophilia.

However, given the rare nature of the disease and the many studies that are initiated with this

population, low response rates may bias the results of the research. Research with both adult and

pediatric populations often have response rates hovering around the 40% mark, which some

regard as a high response rate (Köiter et al., 2009). For example, Elander et al. (2009) had a

response rate of 37% in a study of health-related quality of life in adults with hemophilia. Köiter

et al. (2009) found that 65% of their pediatric hemophilia patients agreed to participate in the

study, but only 47% completed all the study materials. Some authors did not list their response

rate at all (e.g. Gringeri et al., 2004) and many studies failed to provide information on the

patients who declined to participate (e.g. Hassan, Badr, Fattah, & Badawy, 2011; Lindvall et al.,

50

2006). Without such information it was not clear if the non-responders differed in a systematic

way from the study participants.

Convenience sampling methods were often used, with patients having been recruited through

outpatient clinic appointments (e.g. Iannone et al., 2012; Köiter et al., 2009; Van Genderen et al.,

2012). Only a small number of studies were conducted using random sampling methods (e.g.

Hassan et al., 2011; Kellerman et al., 1980; Sherlock, O’Donnell, White, & Blake, 2009), which

would eliminate sampling bias. However, by only inviting patients who are receiving care at

comprehensive care treatment centers, authors have inadvertently excluded patients who may be

unable to afford or are too far away to access care. One study estimated that they were able to

capture 90% of the hemophilia population in that age category at their treatment centers (Köiter

et al., 2009). However, the impact of hemophilia on the lives of that unknown 10% without

access to care may be markedly different from those who receive comprehensive care.

Furthermore, capturing such a large percentage of patients with hemophilia in treatment centers

may be unique to Western Europe, North America, and other developed countries. In developing

countries, there may be many more patients without access to comprehensive care that may go

unstudied. This may impact the generalizability and external validity of the studies, but

accessing an untreated population may prove to be extremely difficult.

Very large studies often require a very large age range of participants, which may not be

appropriate for the topic of study (e.g. Plug et al., 2008).

Another limitation to the current body of research is the lack of a gold standard to measure

quality of life despite the large focus on this construct (Gringeri & Von Mackensen, 2008).

There are generic measures, which are designed for use with persons with various health

conditions, including the general population (Globe et al., 2009). Since a wide range of persons

can be measured with these types of instruments, comparisons can be made across different

populations but these measures may lack the sensitivity to detect subtle changes (Globe et al.,

2009). On the other hand, disease-specific measures are created for use with one population and

can focus on those issues that are most important or relevant to that population, which enhances

the instruments sensitivity to change (Globe et al., 2009). The drawback of using disease-

specific measures is that one cannot use them to compare different populations.

51

The Short Form 36 (SF-36) and the European Quality of Life (EQ-5D) were the most widely

used instruments in a literature review by Fischer et al. (2003) and both are generic measures.

Disease-specific measures of quality of life for children such as the Haemo-QoL and the

Canadian Hemophilia Outcomes-Kids’ Life Assessment Tool (CHO-KLAT) have emerged in the

last few years (Gringeri et al., 2004; Young et al., 2006) but are not widely used in participation

research. Furthermore, few studies define quality of life and how the instruments they use

measure that construct. The variability and the vagueness of the quality of life construct makes

comparison of studies a challenge.

The majority of studies with hemophilia patients are cross-sectional designs (e.g. Elander,

Robinson, & Mitchell, 2009; Hassan et al., 2011; Khair et al., 2011; Sherlock et al., 2009; Van

Genderen et al., 2012), thus causality cannot be inferred. Although a cause and effect

relationship cannot be determined with this study design, cross-sectional studies are favorable in

the hemophilia population because they reduce patient burden and may make a study more

attractive to potential participants, possibly increasing response rates.

During this literature review, a paucity of literature was found regarding self-perception and self-

esteem in boys with hemophilia. Although many studies have used single measures to describe

their population, few studies have critically examined the self-perception and self-esteem of this

population. Articles often allude to an assumption that children with hemophilia are at greater

risk for low self-esteem than their healthy peers (e.g. Thomas & Gaslin, 2001), however during

this literature review only a few articles were identified in the English language that offered a

comprehensive discussion of self-esteem or self-perception in boys with hemophilia.

2.7 Summary

Although there has been much recent research on participation, a large proportion of the studies

revolved around sport participation. This focus on sport may have erroneously resulted in

categorizing children who did not enjoy sports as not participating like their peers. However,

that child may have enjoyed music or art and had a fulfilling life that did not include sport. Thus

it seems apparent that any research focused on participation should take into account all aspects

of participation.

52

Despite many studies assessing self-perception in boys with hemophilia, none of the studies that

were reviewed explored the relationship between self-perception and social participation. Most

recent research seems to indicate that boys with hemophilia in the developed world have

adequate self-perception. Having feelings of self-worth and competency may factor into one’s

social participation and this relationship should be further explored.

Furthermore, participating on par with one’s peers involves participating with one’s peers.

Therefore research into the relationship between social support and social participation is

necessary. Studies have shown that patients with hemophilia may have feelings of isolation

from, or be bullied by, their peers. These interactions with their peer group and the resulting

perception of social support could have an impact on their participation.

Finally, as one’s parents may impose many of the restrictions on one’s participation, it is

important to study the impact of hemophilia on the patient’s family. Studies have shown that the

illness can have a significant impact on the family members, especially the mothers.

Understanding how one’s disease impacts the family may provide insight into one’s social

participation.

The above review of the literature clearly demonstrates that hemophilia is a serious chronic

illness that can have an impact on one’s social participation, self-perception, social support, and

family. The relationships between these factors warrant further exploration.

53

Chapter 3 Methods

3 Methods

3.1 Research Aims

The objective of this study was to examine social participation as a component of quality of life

in children and adolescents with hemophilia and some of the personal and environmental factors

that may contribute to it. Using the ICF framework, it was theorized that the personal factor self-

perception and the environmental factors of social support and disease impact on the family

might be associated with social participation.

3.2 Research Question

The research question posed was: in boys between the ages of 7-18 years with moderate and

severe hemophilia (A and B) from a Toronto-based comprehensive care center, are higher levels

of self-perception, social support, and lower levels of impact on the family associated with

higher social participation?

3.3 Rationale

For the purposes of this study, social participation and its relationship to the personal factor self-

perception and the environmental factors social support and impact on the family were

examined. Self-perception was selected because previous research linked hemophilia with lower

self-perception (Hegeman et al., 2011) and because it has been theorized that lowered self-

esteem could lead to a disrupted social life (Agle, 1984). Social support was chosen because it

was rationalized that if a participant felt he had support from his family and friends, he would be

more likely to participate socially. Likewise, if the participant’s hemophilia had a large impact

on the family, it was believed that social participation might be impacted.

3.4 Significance

The clinical significance of this study lies in understanding what contributes to social

participation. By understanding what factors are associated with social participation, healthcare

professionals may be better informed in their efforts. Given the multiple potential influences of

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social participation, healthcare providers and family members may be able to determine how best

to encourage the patient. Healthcare providers have an opportunity to improve social

participation and by identifying predictors of social participation, healthcare providers may be

better able to identify which of their patients could best benefit from increased support regarding

social participation.

Social participation research in the hemophilia population is very limited and is often focused on

sport participation in children or workforce integration in adults. This is the first step to

understanding social participation in children with hemophilia. By expanding knowledge about

social participation in boys with hemophilia, this study will facilitate and inform program

planning, monitoring, and evaluation, including the assessment of interventions regarding social

participation (van Brakel et al., 2006).

3.5 Hypotheses

The primary hypothesis theorized that there was an association between self-reported self-

perception and social participation. As per the ICF model, the relationship between these

variables should be bidirectional in nature. Participants who have higher levels of self-worth or

self-competence may feel more at ease participating in social activities or may be invited to

participate in social activities more readily (Köiter et al., 2009; Petrini & Seuser, 2009).

Furthermore, participants who participate more may feel better about themselves. Thus, a

participant who reports higher levels of self-perception may correspondingly report higher levels

of social participation, while participants with lower levels of self-perception may report lower

levels of social participation. Given that the relationship is bidirectional, the reverse is also true

such that participants who report higher or lower levels of social participation will

correspondingly report higher or lower levels of self-perception.

The secondary hypothesis was that the variables perceived social support and social participation

were associated in a bidirectional manner. If a participant feels supported by parents, teachers,

classmates, and close friends, they may also tend to engage in more social participation (Coppola

et al., 2011). Therefore, participants with greater perceived social support will endorse higher

social participation, while participants with lower levels of perceived social support will endorse

lower levels of social participation. Again, the reverse may be true such that participants with

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higher or lower levels of social participation will report correspondingly higher or lower levels of

perceived social support.

The third hypothesis posited that there was an association between the impact on the family and

social participation. If parents or guardians report greater negative impact of the participant’s

disease on the family, the participant’s reported social participation may also be lower. The

bidirectional nature of the relationship means that the reverse may also be true: participants who

report higher social participation may have parents who report correspondingly fewer negative

impacts of the participant’s disease on the family. For example, if a parent reports feeling very

worried about their child engaging in physical activity, the participant may not be permitted to

play sports. If the participant does engage in a high impact sport, the parent may worry very

much about the child sustaining injuries.

3.6 Study Design

The project was a substudy of a fully funded, registered clinical study. Although this study was

embedded in a larger international study, the decision to study social participation was mine. A

methodologist, a psychologist, a hematologist, and myself contributed to the study design and the

selection of measures. I developed the study design with supervision and advice from my thesis

committee members and thus this thesis refers to the work that we collectively contributed to.

A cross-sectional observational study was conducted at The Hospital for Sick Children. Since

the hemophilia population is small and highly studied, cross-sectional studies minimize

participant burden and maximize the number of studies participants may be willing to participate

in. Cross-sectional designs are often employed in hemophilia research (e.g. Gringeri et al., 2004;

Hassan et al., 2011; van der Net et al., 2006). This design minimized participant dropout

because there was no follow-up period where participants could be lost. The inability to make

causal inferences is a disadvantage of this study design, however the exploratory nature of the

research question nevertheless makes this an attractive design. Furthermore, the ICF model

suggests that any relationship found between the variables will be bidirectional in nature. Cross-

sectional studies are favorable in the hemophilia population because it reduces participant burden

and may make a study more attractive to potential participants, possibly increasing response

rates.

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Figure 2 illustrates the study design used for this research study.

Figure 2. Study Design

3.6.1 Ethical Issues

The original clinical trial, of which this project was a substudy, obtained ethics board approval

from The Hospital for Sick Children on December 2, 2010. This study was added to the existing

clinical trial to prevent oversaturation of the patient population. An amendment to incorporate

this study’s measures was submitted to The Hospital for Sick Children’s Research Ethics Board

and approval was obtained April 21, 2011. Research Ethics Board administrative approval from

the University of Toronto was obtained May 24, 2012, allowing for time to develop the substudy

protocol (see Appendix A).

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This study was carried out in accordance with the Declaration of Helsinki. As such, a research

staff member reviewed with all research subjects the purpose, procedures, potential benefits,

potential harms, discomforts, or inconveniences; and confidentiality of the research study prior to

consent. Subjects were informed that refusal to participate in the research study would not affect

their future care at The Hospital for Sick Children.

All subjects above the age of 11 were asked to complete a written informed consent form

approved by The Hospital for Sick Children’s Research Ethics Board.

All subjects under the age of 11 were asked to give assent, while the parent/guardian of the

subject was asked to complete an approved written informed consent form regarding the

recruitment of the subject. If the subject refused to participate in the research study the patient

was not recruited, even if the parent/guardian gave consent.

All parents/guardians who completed a questionnaire signed an informed consent form allowing

for the data collected in the study to be used for research purposes.

3.6.2 Participants

The Hospital for Sick Children’s Comprehensive Care Hemophilia Program, located in Toronto,

Ontario, Canada, draws from a large population base and services approximately 180 hemophilia

patients in the central Ontario region. A representative sample of the hemophilia population can

be drawn from this population, as the Program sees all patients with hemophilia living in both

urban and rural areas within this region. The standard of care in Canada is to routinely

administer or prescribe prophylaxis at the treatment center (Kern et al., 2004). Given that this

hemophilia treatment center follows these standards, the results of this study will likely have

generalizability to other clinics in similar settings (e.g. the United States and Western Europe).

It is important to bear in mind that this research was conducted with hemophilia patients in

Canada. All hospital and physician services, including factor concentrate and supplies, are

covered by the provincial healthcare plan, which has no insurance caps. Similar plans can be

found throughout Canada and thus our sample is a snapshot of Canadian hemophilia patients.

That being said, this research study was conducted in Toronto, Ontario, Canada, which is a

multicultural city with a higher rate of immigrants (49%) than the rest of Canada (21%) (2011

National Household Survey, 2013).

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Table 1 details the inclusion and exclusion criteria for participation in this study. Boys with

moderate and severe hemophilia A and B between the ages of 7-18 were the population of

interest. For patients with severe hemophilia, trauma as well as spontaneous bleeding can lead to

hemorrhages that may occur as often as several times a month; moderate hemophilia patients

bleed less often, usually in response to trauma (Carcao, 2012; Franchini et al., 2012). Patients

with mild hemophilia have fewer hemorrhages and may not be as severely impacted by their

disease, thus they were not part of the population of interest.

Although recent evidence suggests that hemophilia B may have a less severe bleeding phenotype

and better long-term outcomes than hemophilia A (Franchini et al., 2012), traditionally

hemophilia A and B have been considered to have similar clinical symptoms and signs (Carcao,

2012). For the purposes of this study, types A and B were analyzed jointly as the population

under examination was younger.

Patients between the ages of 7-18 were the population of interest because development in social

dimensions becomes more prominent in older children and teenagers (Gringeri et al., 2004). As

patient self-report is the gold standard for measuring perceived health-related quality of life

(Varni, Limbers, & Burwinkle, 2007), excluding patients younger than 7 years of age who may

need to respond by proxy likely increased the accuracy of the results.

Participants were categorized as children or adolescents based on their age at the time of the

study. The World Health Organization defines adolescence as the period after childhood and

before adulthood, from the ages of 10-19 years (World Health Organization, 2014). However,

the instruments used for this study were developed and validated with the following categories:

children were between the ages of 7-12 and adolescents were between the ages of 13-18. Given

that these instruments were validated based on these age categories, we have used the definition

provided by the instruments.

All participants were required to be able to speak in English (with the assistance of a study team

member for either reading or clarification, if needed), as the study questionnaires were only

available in the English language.

Patients with comorbid illnesses noted in their medical chart (juvenile arthritis, muscular

dystrophy, neurologic impairment, cognitive impairment, or other musculoskeletal illnesses)

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were excluded from the study. These illnesses may have independently affected the variables of

interest if the distribution of one variable was systematically different for patients with comorbid

illness than in patients without. Eliminating potential confounding variables allowed for greater

accuracy in determining relationships, if any, between the variables.

Parents and guardians of participants were invited to complete one questionnaire. Inclusion

criteria were defined as being a parent or guardian of a participant in the study, able to speak in

English (with the assistance of a study team member for either reading or clarification, if needed)

and above the age of 18.

Given that the hemophilia population is so highly studied, a sample of patients was selected for

recruitment. From a list of all eligible patients receiving care at The Hospital for Sick Children,

a sample was generated using an electronic randomization program, stratified by severity of

illness (moderate versus severe) and age (7-12 years versus 13-18 years). The sample was

further weighted to reflect the larger proportion of patients with severe disease (34 severe

patients, 16 moderate patients).

By choosing a random sample, sampling bias was eliminated that may have arisen through

convenience sampling in outpatient clinics. The randomization was not used to divide the

sample into groups for comparison, but rather to gather a representative sample by eliminating

the likelihood that the sample differed systematically from non-participants.

Table 1. Inclusion and Exclusion Criteria Inclusion Criteria Exclusion Criteria Participants • 7-18 years of age

• Hemophilia A or B • Moderate or severe phenotype • English speaking

• Comorbid illness

Parents/Guardians • 18 years of age or above • English speaking • Parent and/or guardian of participant

in study

• Cognitive impairment

3.6.3 Sample Size

Table 2 is a sample size table that was calculated to determine the study sample size needed for

moderate correlations between variables. Cells in the table indicate the 95% confidence interval

given the specified sample size. To detect a moderate correlation (r = 0.4), a sample of 50

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subjects provided a statistically significant confidence interval that ranged from 0.1 to 0.6 (i.e.

differentiates from low or high correlations).

Table 2. Sample Size Sample Size (n) 10 20 30 40 50

Corr

elat

ion

(r)

0.2 -0.5-0.7 -0.3-0.6 -0.2-0.5 -0.1-0.5 -0.1-0.5

0.4 -0.3-0.8 -0.1-0.7 0.0-0.7 0.1-0.6 0.1-0.6

0.6 -0.0-0.9 0.2-0.8 0.3-0.8 0.4-0.8 0.4-0.8

0.8 0.3-1.0 0.6-0.9 0.6-0.9 0.7-0.9 0.7-0.9

3.6.4 Recruitment

An introductory letter approved by the institution’s Research Ethics Board was sent to the

families of all eligible patients on the sample list generated by the electronic randomization

program. The hematologist or other clinic staff in the patient’s circle of care approached patients

by telephone or in clinic until all required study participants were recruited to the research study.

A research staff member met with families who agreed to participate and gathered informed

consent. Patients who gave consent or assent and whose parents and/or guardians gave informed

consent were subsequently enrolled in the study.

3.6.5 Administration

Participants were recruited and participated in the study between June 2011 and October 2013.

Study measures were either answered using self-report or administered in an interview fashion.

For self-report questionnaires, research staff was available if the participants or parents/guardians

required assistance completing the questionnaires. For questionnaires administered in an

interview fashion, a question-by-question guide was developed to ensure standardized

completion of questionnaires.

The demographics and the health information sheets were completed by clinic staff and by the

participant’s parents/guardians.

The Pediatric Quality of Life™ Family Impact Module was completed by the participant’s

parent/guardian. All other self-report questions were directed to participants.

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3.7 Data Collection Instruments

The data collection instruments selected for use in this study were carefully chosen by the study

team based on empirical evidence validating their ability to measure self-perception, perceived

social support, impact on the family, and social participation (see Appendix B).

Measures that collect self-reported data were chosen as they offered insight as to how the

participants viewed their own situation. Although it may be less objective than other data, it

allowed for the opportunity to gather data that may be unavailable through a medical chart or

laboratory test.

For the larger study of which this study was a substudy, data was collected from each of the ICF

domains by the instruments indicated in Table 3.

Table 3. Data Collection Instruments ICF Domain Instrument Health Condition • Health information sheet

• Physician interview Body Functions and Structures • Hemophilia Joint Health Score

• Radiographs Activity • Functional Independence Score in Hemophilia

• Activity Scale for Kids Participation (Social) • Participation Scale for Kids

• Canadian Hemophilia Outcomes-Kids’ Life Assessment Tool • Pediatric Quality of Life Inventory™

Environmental Factors • Patient demographics sheet • Social Support Scale for Children • Pediatric Quality of Life™ Family Impact Module

Personal Factors • Patient demographics sheet • Self-Perception Profile for Children or Self-Perception Profile for Adolescents

This substudy was focused on the information collected from the domains of health condition,

participation (social), environmental factors, and personal factors.

3.7.1 Demographics

The demographics sheet collected information on participant’s age at assessment (years),

ethnicity, type of hemophilia (A versus B), factor level (mild, moderate, or severe phenotype),

school attendance, school grade, primary guardian, parental marital status, living area (rural

versus urban), and annual household income (low [<$30,000], medium [$30,000-$80,000], or

high [>$80,000]).

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3.7.2 Health Information

The health information sheet collected information on the age at which infusions began (years),

age at which self-infusion started (if applicable), who infuses the treatment (patient, mother,

and/or father, clinic staff), where treatment takes place (home, clinic, emergency room),

frequency of treatment in the last six months (on-demand, once per week, 2 times per week, 3

times per week, every other day, every day, immune tolerance induction or bypassing agents),

and frequency of treatment over the patient’s entire life (on-demand, primary prophylaxis,

secondary prophylaxis, and/or short-term prophylaxis).


Social participation was measured using a modified version of the Participation Scale. It is a

generic measure intended for use by people with a wide variety of conditions, thus there are no

disease-specific questions on the scale (van Brakel et al., 2006). The original Participation Scale

was intended for adults and was modified with the author’s approval for this study to be relevant

to North American children.

One appealing aspect of the Participation Scale was that it did not merely measure the subject’s

participation in an activity or social situation; rather it measured the impact of non-participation.

The Participation Scale accounts for non-participation in activities that are irrelevant or of no

interest to the subject. This scale follows the recommendations of Gill and Feinstein (1994) that

suggest quality of life measurements should have participants rate the severity and the

importance of the domains that they are measuring. Lack of participation may reflect limitations

in opportunity or in one’s ability to participate in an activity or social situation, but it may also

reflect a lack of interest in said activity. Therefore it was critical that the instrument used for

social participation measured relevance as well.

Items on the scale prompted subjects to report if they participated as much as their peers did in

an activity. Peers were defined as “those who are similar to the respondent in all respects (socio-

cultural, economic and demographic) except for the disease or disability” (van Brakel et al.,

2006). If they reported to participate as much as their peers did, the item was scored 0. If they

found the activity to be irrelevant, to be something that they did not have to do, or something that

they did not want to do, the item was also scored 0. However, if the subject responded that they

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did not participate as much as their peers, or that they only participated as much sometimes, the

item was scored from 1 to 5, with 1 indicating no self-perceived problem and 5 indicating a large

problem with social participation. Higher scores on the measure indicated greater problems with

social participation as compared to one’s peers.

This scale was modified because it was felt at the time the study was conducted there were no

adequate measures of social participation specifically designed for children. With permission by

the author, the modified measure was re-named the Participation Scale for Kids and several

questions were modified to be relevant to children as opposed to adults. The comparison to

one’s ‘peer’ was changed to ‘classmate’ for ease of understanding and with the assumption that

in this sample most participants attended school. For example, the original Participation Scale

asks respondents, “Do you work as hard as your peers do (same hours, type of work, etc.)?” This

item was modified for children to ask, “Do you work as hard as your classmates do (same hours

of school-work, type of work, etc.)?” The peer comparison of ‘classmate’ was chosen over

‘friend’ as it was theorized that participants would more likely be friends with someone who had

similar levels of participation and may not reflect the peer group the original measure intended.

For example, participants may be friends with other children with chronic illness and they may

all have reduced levels of social participation. They would all indicate on the measure that they

participated as much as their friends did, even though they did not participate at the same level as

their peers.

The Participation Scale was further modified by adding questions that may be relevant to

children in North America such as, “Do you play games as often as your classmates do? (e.g.

video games, board games, etc.)” All changes to the final 23-item scale were discussed with the

original author of the Participation Scale and permission to use the modified scale was granted

(W. H. van Brakel, personal communication, April 23, 2011).

The original 18-item Participation Scale for adults had a maximum score of 90 and the grades of

participation restriction were as follows: no significant restriction (0-12), mild restriction (13-

22), moderate restriction (23-32), severe restriction (33-52), and extreme restriction (53-90) (van

Brakel et al., 2006). Given the increased number of items on the Participation Scale for Kids

which allowed for a maximum score of 115, grades of participation restriction were

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proportionately re-defined as: no significant restriction (0-15), mild restriction (16-27), moderate

restriction (28-40), severe restriction (41-66), and extreme restriction (67-115).

Additionally, three open-ended questions were included probing subjects for their thoughts on

what participation consisted of, the effect (if any) of hemophilia on their participation, and the

effect (if any) of personality on participation. The additional questions are listed in Table 4.

Table 4. Participation Scale for Kids Questions (Open-Ended) Question 1. What do you think participation is? 2. Do you feel that hemophilia stops you from participating like others? If so, how? 3. Do you feel that personality changes how you participate with others? (e.g. your thoughts, feelings, and behaviors.)

The primary reason why this measure was chosen for this study was because for every item on

the scale, there was an option to indicate the relevancy of the activity to the participant. The

measure has good face validity and seemed relevant to the study. Also, this instrument was

designed based on the participation domains of the ICF, was cross-cultural in nature, aimed to

assess patient-perceived participation, and it was not disease-specific (van Brakel et al., 2006).

The original scale had high internal consistency (Cronbach’s α 0.9) and high inter-tester

reliability (0.8) (van Brakel et al., 2006). The authors felt that there were no existing tools that

could be used to validate this instrument under the prevailing conditions, thus they chose to

validate the results against expert opinions. They found moderate correlations between the

expert opinions and the scores of participants (rho = 0.4).


Self-perception was measured using items from the Self-Perception Profile for Children or the

Self-Perception Profile for Adolescents (Harter, 1985a; 1988; 2012a; 2012b), often considered

the gold standard for measuring self-perception. Five specific domains of perceived competence

are tapped by the original measure: Scholastic Competence, Social Acceptance, Athletic

Competence, Physical Appearance, and Behavioral Conduct. A sixth subscale measures the

child or adolescent’s perception of their Global Self-Worth. There are two versions of the scale:

one for children (ages 7-12) and one for adolescents (ages 13-18). By having different versions,

the scales ensured that questions were relevant to the participant’s age group.

The questionnaire first asked participants to choose between two statements describing

conflicting types of kids or teenagers, e.g. “some kids have a lot of friends” or “other kids don’t

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have very many friends.” Once the participant made his choice, he then had to indicate if the

chosen statement was ‘really true for me’ or ‘sort of true for me.’ Items on each of the subscales

were scored 4, 3, 2, or 1; higher scores indicated higher perceived competence on that item.

There was no overall score for the scale as the measure yielded six subscale means. Within each

subscale, the items were counter-balanced to avoid participants yea-saying or acquiescing when

filling out the questionnaires (Streiner & Norman, 2008a).

To reduce burden on participants, three of the six domains were selected for inclusion in this

study. The questionnaire manuals note that some subscales can be lifted from the instrument as

long as all items of the given subscales are administered to the participant (Harter, 2012a;

2012b). Other researchers have successfully administered individual subscales to their samples

(e.g. Crocker, Sabiston, Kowalski, McDonough, & Kowalski, 2006; Crockett, Moilanen,

Raffaelli, & Randall, 2006). The three domains chosen were Social Acceptance, Athletic

Competence, and Global Self-Worth. These domains were chosen as they had the most face

validity suggesting a relationship with social participation. The domain of Social Acceptance

measured the extent to which the child felt he was accepted by his peers or felt popular, Athletic

Competence examined the child’s competence in sports and outdoor games, and Global Self-

Worth tapped into the degree to which the child liked himself as a person (Harter, 1985a). These

three domains yielded an 18-item measure for children and a 15-item measure for adolescents.

The Self-Perception Profile for Children and the Self-Perception Profile for Adolescents were

chosen for several reasons. Firstly, they have been used extensively in the literature, including

studies with boys with hemophilia (e.g. Hegeman et al., 2011; Trzepacz, Vannatta, Davies,

Stehbens, & Noll, 2003). Secondly, the question format of the measures were designed to off-set

the tendency for children to give socially desirable responses (Harter, 1988). The way the items

are designed there is no ‘right’ or ‘wrong’ choice, only one that is truer for the participant than

the other. Third, this measure demonstrated strong internal consistency when administered to

four separate samples in North America (Harter, 1985a). For the child version, Cronbach’s α

was 0.8-0.8 for Social Acceptance, 0.8-0.9 for Athletic Competence, and 0.8-0.8 for Global Self-

Worth, depending on the sample (Harter, 1985a; 1988). The same process for determining the

psychometric properties of the adolescent scale was used and revealed internal consistency to be

high as well (Cronbach’s α 0.8-0.9 for Social Acceptance, 0.9-0.9 for Athletic Competence, and

0.8-0.9 for Global Self-Worth). Lastly, the scales provided reference scores for four samples of

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North American children and adolescents. These samples were drawn from lower middle class

to upper middle class neighborhoods and were primarily composed of Caucasians (Harter, 1988).


The Social Support Scale for Children is a self-report tool that was used to measure perceived

support and positive regard from significant others in a participant’s life. Four sources of social

support or positive regard were tapped in this scale: parents, teachers, classmates, and close

friends. These sources were included in the measure because the opinions of these sources may

influence the self (Harter, 1985b). The parent subscale examined to what extent the parents of

the child understood or cared about them, the teacher subscale asked to what degree did one’s

teacher help him achieve his best and cared about him, the classmate subscale assessed if the

child perceived that his classmates liked him the way he was or asked him to join in play, while

the close friend scale asked if the child had a close friend who understood him or who listened to

him. All of the items were tapping into the positive regard from others that was perceived by the

respondent.

Like the Self Perception Profiles for Children and Adolescents, this measure required

respondents to choose between two opposing statements and then identify if the statement was

‘really’ or ‘sort of’ true for them. For example, the respondent may have had to choose between

“some kids have classmates who like them the way they are” or “other kids have classmates who

wish they were different” and then indicated how true that statement was for them. Items on

each of the subscales were scored 4, 3, 2, or 1, were counter-balanced, and higher scores

indicated more perceived social support and positive regard. There was no overall score and the

scale yielded four subscale means. All the subscales of this measure were included in this study.

The Social Support Scale for Children was chosen primarily for its similarity to the Self-

Perception Profiles for Children and Adolescents. Having scales that were rated in a similar

fashion made it easier for participants to complete the battery of assessments. As the initial

intent of the measure was to determine the forms of social support that would best predict a

person’s global self-worth, its use in this study was quite fitting. The scale also had strong

internal consistency (Cronbach’s α 0.7-0.9). As with the Self-Perception Profile for Children

and Adolescents, reference scores are provided from four samples drawn from lower middle to

upper middle class neighborhoods (90% of whom were Caucasian) (Harter, 1985b).

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3.7.6 Impact on the Family

Hemophilia’s impact on the family was assessed using the Pediatric Quality of Life (PedsQL™)

Family Impact Module. This measure strived to assess the impact of a pediatric chronic health

condition, such as hemophilia, on the family members of the participant in the previous month.

It had eight subscales measuring physical functioning (e.g. feeling tired), emotional functioning

(e.g. anxiety), social functioning (e.g. feelings of isolation), cognitive functioning (e.g. difficulty

maintaining attention), problems with communication (e.g. communicating with healthcare

professionals), problems with worry (e.g. worrying about treatments), problems with daily

activities (e.g. activities taking more time and effort), and problems with family relationships

(e.g. stress) (Varni, Sherman, Burwinkle, Dickinson, & Dixon, 2004). The measure calculated

three summary scores based on the dimension scores: the Total Score, the Parent HRQoL

Summary Score and the Family Functioning Summary Score.

Respondents answered items on a scale ranging from 0 (‘never a problem’) to 4 (‘almost always

a problem’) and the items were subsequently reverse-scored and linearly transformed to a 0-100

scale such that higher scores on the scale indicated less negative impact on the family and better

family functioning (Varni et al., 2004). To obtain the Scale Scores, the sum of the items was

divided by the number of items answered by the participant to account for data that may be

missing.

This measure was selected for inclusion in this study partially because the PedsQL™ Inventory

was being used in the larger study of which this study was a substudy. Furthermore, the large

number of domains tapped by this scale allowed us to examine the varying ways in which

chronic disease impacted the family. This measure was also chosen because its internal

consistency was high for all the scale scores with Cronbach’s coefficient α = 1.0 for the total

impact score (all items), 1.0 for the Parent HRQoL Summary Score, and 0.9 for the Family

Functioning Summary Score.

3.8 Data Management

All data was manually entered into either Microsoft Excel 2003 or FileMaker Pro 11 in

accordance with the database-coding manual. The database-coding manual ensured that data

was entered in a standardized fashion and was developed to prevent coding errors. Data entered

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in FileMaker Pro 11 was exported to Microsoft Excel 2003 and subsequently imported to

statistical analysis software.

Double data entry of 100% of the data was performed to ensure the fidelity of the data.

All data was stored in keeping with hospital privacy policies and will be destroyed as required by

hospital policy.

3.9 Analysis

SAS version 9.2 was used for statistical analysis.

For all tests, statistically significant differences were established if two-tailed p-values were <

0.05.

3.9.1 Participation Scale

To test the reliability of the modifications made to the Participation Scale, internal consistency

was tested using Cronbach’s coefficient alpha. This test revealed the homogeneity of the items

that formed the scale and could be used when there had only been a single administration of the

test (Streiner & Norman, 2008b). An α of 0.7 is the minimum recommended by Nunnally (1978)

for research purposes (as cited in Streiner & Norman, 2008a).

3.9.2 Missing Data

For values that were missing, a variety of techniques were used to account for the data.

3.9.2.1 Single Imputation

The Participation Scale Users Manual did not contain instructions on how to handle data that was

missing. However, psychometric testing of the Participation Scale by the original scale’s author

assigned a mean score of the participants’ completed items to missing items in the Participation

Scale (Stevelink, Terwee, Banstola, & Van Brakel, 2012).

For missing items on the Self-Perception Profile for Children and the Self-Perception Profile for

Adolescents, the author recommended that a subscale mean be calculated by summing the scores

of the completed items and dividing by that number of items to obtain a prorated subscale

average (Harter, 2012a; 2012b). This method was only recommended if the participant answered

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at least four of the six items for that subscale (Harter, 2012b). Although the subscales for the

Self-Perception Profile for Adolescents only contained five items each, we still used a cut-off of

at least four items being answered to calculate a subscale score.

The manual for the Social Support Scale for Children does not specifically detail how to handle

missing data, however given that the format of the scale is identical to that of the Self-Perception

Profile for Children and the Self-Perception Profile for Adolescents, we used the same method

advised by the author to obtain prorated subscale averages and only calculated subscales if four

of the six items were answered per subscale.

The authors of the PedsQL™ Family Impact Module recommended that scale scores be

computed as the sum of the items divided by the number of items answered to account for

missing data (Varni et al., 2004). However, if more than 50% of the items were missing, a scale

score was not to be computed.

Although this method of dealing with missing data was recommended by all of the authors of the

outcome measures, this method had some limitations. Mean imputation reduced the standard

deviation because it was highly unlikely that the values that were missing from the data were

exactly equal to the mean. If the standard deviation was reduced, the standard error would be

reduced as well, which could have artificially inflated the probability of finding a significant

difference between groups (Norman & Streiner, 2003).

3.9.2.2 Sensitivity Analysis

To test the robustness of the primary analysis and the use of single imputation, we conducted a

sensitivity analysis. Analysis was run to compare the worst- and best-case scenarios with the

single imputation results to see if the results were sensitive to change. Using the minimum and

maximum values for missing variables may have yielded values that were too extreme to be

considered reasonable for the missing values. Thus for the worst-case scenario, missing data was

imputed as the 10th percentile value and for the best-case scenario, the 90th percentile value.

3.9.3 Demographics

Descriptive statistics were used to describe the sample, depicting the age, type of hemophilia,

factor level, ethnicity, school attendance, school grade, primary guardianship, parental marital

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status, living area, household income, primary infuser, age infusions began, age self-infusion

began (if applicable), place of infusion, and frequency of infusion in the last six months.

Quantitative variables were expressed as means plus or minus standard deviations, while

qualitative variables were expressed as counts and proportions.

3.9.4 Correlation Coefficients

To assess if there was a relationship between the variables of interest, Pearson product-moment

correlation coefficients were calculated. Pearson correlations measured the linear relationship

between two variables (Zou, Tuncali, & Silverman, 2003). Rough guidelines for interpreting the

strength of the relationship vary but the most widely known operational definitions for

interpreting correlation coefficients magnitude are as follows (in absolute values): ≤ 0.1

represents small correlations, 0.3 are medium correlations, and 0.5 to 1.0 are large correlations

(Hemphill, 2003). Relationships were assessed between the total score of the social participation

scale, each of the subscale means for self-perception, the subscale means for social support, and

both the total and the dimension scores for impact on the family.

3.9.5 Independent Samples t-tests

Differences between groups were assessed using independent samples t-tests, which measured

the probability that the differences could have arisen by chance (Norman & Streiner, 2003).

Groups of interests were the children versus adolescent participants in this sample. Differences

between this sample and reference groups provided for self-perception and social support were

also assessed.

3.9.5.1 Model Checking

The use of Pearson correlations is dependent on data meeting several assumptions.

Homoscedasticity is the assumption that is of utmost importance and residual plots were

constructed to observe the relationships between the residuals.

In addition to data being homoscedastic, data should also be of an interval or ratio scale,

variables must be approximately normally distributed, a linear relationship must describe the two

variables, and outliers must be minimized (De Veaux, Velleman, Bock, Vukov, & Wong, 2012).

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Although all of the instruments used appear to be rated on ordinal scales, for the Self-Perception

Profile for Children, the Self-Perception Profile for Adolescents, the Social Support Scale for

Children, and the PedsQL™ Family Impact Module it is the mean of the items that is used for

analyses. The Participation Scale for Kids also appears to be ordinal but it is scored by

summation of all items. The correlation coefficient can be used to investigate associations

between two interval or ordinal variables (McCrum-Gardner, 2008), thus the use of Pearson’s

product-moment correlation coefficients are appropriate.

The normality of residuals was determined using the Shapiro-Wilk test given that the sample size

was less than 2000. If the residuals were not normally distributed, it may have suggested that a

non-linear relationship best described the relationship. Test results for normality should have

been non-significant to indicate normality.

The assumption was that there were no influential outliers in the Pearson correlation coefficients

and this was verified by using casewise diagnostics. Each datapoint was removed in sequence to

evaluate its effect on the regression line and was verified with DFFITS.

In the event that the data did not meet the assumptions required to warrant use of Pearson

correlation coefficients, Spearman rank-order correlation coefficients were to be used. This

allowed us to identify any changes to the correlations observed should the data be non-

parametric. Data must meet two assumptions to use Spearman’s rank-order correlation: data

must be ordinal, interval or ratio, and must reflect a monotonic relationship. As previously

discussed, the data appear to be ordinal and thus meet the first assumption. Scatter plots were

used to assess if the data reflected a monotonic relationship, which occurs when an increase in

one variable results in an increase or decrease of another variable.

3.9.6 Multiple Linear Regressions

As this study was cross-sectional in nature, directionality of any of the variables observed cannot

be concluded. However, multiple linear regressions were constructed to explore the amount of

variation in social participation possibly explained by self-perception and social support. One

cannot exclude the possibility that the inverse may be true: that it is in fact social participation

that influenced self-perception or social support. The purpose of the multiple linear regressions

was to explore the data further.

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The model was evaluated for overall significance with an omnibus F-test and individual

covariates were further evaluated for significance.

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Chapter 4 Results

4 Summary of Results

4.1 Introduction

The results of this study showed that boys with hemophilia in Toronto, Canada, had minimal

social participation restrictions, high levels of perceived competence, and perceived that they had

high levels of social support. There appeared to be a relationship between social participation

and self-perception as well as social participation and social support. No important relationship

was found between social participation and impact on the family.

4.2 Enrolment

The use of an electronic randomization program to generate a sample of patients to be

approached for the research study ensured that the sample was highly generalizable.

Based on the electronically generated sample, 61 patients were assessed for eligibility and 57

(93.4%) were deemed eligible to participate. Those who were excluded from the study were

excluded due to age and language barriers (n = 4).

Clinic staff approached the 57 eligible patients and their parents/guardians to participate in the

study and 51 (89.5%) gave informed consent/assent as appropriate. Fifty of 51 consented

participants completed all assessments; one participant consented to participate but was unable to

attend the clinic for assessments and was subsequently withdrawn from the study. The final

sample included 50 assessable participants and a corresponding 50 assessable parents/guardians.

4.3 Participant Characteristics

Demographic data and characteristics of the participants are listed in Table 5.

The sample included participants with hemophilia whose age ranged from 7.7 to 17.0 (M = 12.1,

SD = 2.8). Thirty children (aged 7-12) and 20 adolescents (aged 13-18) participated in the study.

The sample was composed predominantly of participants with hemophilia A and 70% of

participants had severe hemophilia.

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When asked to identify their ethnicity, most participants indicated that they were North

American, followed by South Asian, European, and then Middle Eastern. A fifth of the sample

identified their ethnicity as Other, a category generally used when the participant reported two or

more ethnicities.

Forty-nine of 50 (98.0%) participants attended school (one participant was home-schooled,

2.0%) and the school grades ranged from 2 to 12 (M = 6.6, SD = 2.8).

Table 5. Participant Characteristics (N = 50) Variable n (%) Type of Hemophilia

Hemophilia A Hemophilia B

41 (82.0) 9 (18.0)

Phenotype Moderate Severe

15 (30.0) 35 (70.0)

Ethnicity North American Other South Asian European Middle Eastern African Caribbean Chinese Korean Mediterranean South American Vietnamese

16 (32.0) 10 (20.0) 6 (12.0) 5 (10.0) 3 (6.0) 2 (4.0) 2 (4.0) 2 (4.0) 1 (2.0) 1 (2.0) 1 (2.0) 1 (2.0)

4.4 Family Situation

Details of participants’ family situation were collected from the participant’s primary guardian

and are listed in Appendix C.

4.5 Treatment

Parents were asked about the participant’s treatment history, which was verified through medical

charts. Further details of the participant’s treatment history are listed in Appendix D. As

prophylaxis is the standard of care for severe hemophilia patients at The Hospital for Sick

Children, when recounting treatment frequency for the previous six months over two-thirds of

the participants were on prophylaxis (receiving treatment two or three times a week, or every

other day). Of the severe hemophilia patients, 29 (83%) patients were on prophylaxis. Nearly a

quarter of all participants received treatment on-demand.

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There was a wide range of ages at which infusions began with some participants requiring factor

infusions immediately after birth and some not being infused with factor until the age of 15 (M =

1.7, SD = 2.9).

Clinic staff supported the use of self-infusion when appropriate. In this sample, 15 (30.0%)

participants reported they had learned how to self-infuse and the average age that self-infusion

began was 11.2 years old (Minimum = 8.0, Maximum = 15.5, SD = 2.2). Some participants who

reported an age at which self-infusion began only identified a parent as the infuser.

4.6 Internal Consistency

The reliability of the modified Participation Scale for Kids was tested with Cronbach’s alpha (see

Appendix E). Both the overall raw alpha (α = 0.7) and the standardized alpha (α = 0.7) were on

par with the 0.7 cut-off previously specified (Nunnally, 1978, as cited in Streiner & Norman,

2008a).

4.7 Outcomes


The mean score on the Participation Scale for Kids was 6.5 (SD = 6.2). Forty-six (92.0%)

participants had no significant restriction (scores between 0-15), 3 (6.0%) participants had mild

restriction (scores between 16-27), and one (2.0%) participant had moderate restriction (scores

between 28-40). No participants indicated that they had severe restriction (scores between 41-

66) or extreme restriction (scores between 67-115). Item responses for each item are displayed

in Appendix F.

Although the mean score was still within the mild restriction range, some respondents reported

that they had “large problems” feeling comfortable meeting new people (item 17, n = 3) and felt

that they did not have the same respect in the community as their classmates (item 8, n = 3).

Each of the following items were endorsed once by a respondent as being a “large problem,”

though not necessarily by the same respondent:

2) Do you work as hard as your classmates do?

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6) Do you take part in casual recreational/social activities as much as your classmates do?

(e.g. sports, chats, meetings, etc.)

7) Are you as socially active as your classmates? (e.g. in religious/community affairs,

etc.)

13) In your neighbourhood, do you visit public places as often as other people do? (e.g.

schools, shops, tea/coffee shops, etc.)

20) Do you use social networking sites as often as other people do? (e.g. Facebook,

Twitter, Myspace, etc.)

Some participants felt that they had “medium problems” when it came to participating as their

peers. These included being able to take part in casual recreational/social activities (item 6, n =

3), having the same respect in the community (item 8, n = 3), working as hard (item 2, n = 3),

helping other people (item 16, n = 3), playing games as often as their peers (item 19, n = 3), and

visiting other people in the community (item 11, n = 2).

Other items endorsed by at least one participant as a “medium problem” included:

3) Do you contribute to the household financially in a similar way to your classmates?

5) Do you take part in major events as your classmates do? (e.g. weddings, funerals,

religious festivals, etc.)

10) Do you have the same opportunities as your classmates to start or maintain a

relationship?

17) Are you comfortable meeting new people?

Children’s responses on the Participation Scale for Kids elicited a mean score of 7.6 (SD = 7.2)

while adolescents had a mean score that was slightly lower at 4.9 (SD = 3.7). Based on these

scores, all adolescents indicated that they had no significant restriction when it came to

participation. However, when comparing the means of the children and the adolescents,

allowing for unequal variances, the difference between the two groups was not statistically

significant (t(45.4) = 1.8, p = 0.1).

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Participants were asked what they thought participation was and 34 (68.0%) participants said that

it involved being included or involved in an activity. Other responses as to what participation

was included working hard (n = 4, 8.0%), volunteering (n = 3, 6.0%), being allowed to take part

in an activity (n = 3, 6.0%), learning new things (n = 2, 4.0%), cooperating/helping (n = 1,

2.0%), and “what you do” (n = 1, 2.0%). Only two participants (n = 2, 4.0%) replied that they

did not know what participation was and both of these participants were quite young (7.8 and 8.8

years of age).

When participants were asked if their hemophilia prevented them from participating like others,

29 (58.0%) said that hemophilia only prevented them a very little bit (“barely” or “not really”) or

not at all. For the remaining 21 participants (42.0%), hemophilia primarily prevented them from

participating in contact sports and physical activity, particularly hockey and football.

Nearly half (n = 24, 48.0%) of the participants felt that personality did not affect how they

participated with others, but the rest reported that it did. Most of the responses revolved around

how being angry or being in a bad mood could lead to one participating less with others. Some

participants responded that when they participated with others, they felt happier.


Overall, the participants scored highly when it came to self-perception subscales. Given that the

highest score on any of the subscales was 4.0, the mean scores for each of the subscales are

displayed in Table 6.

Table 6. Self-Perception Subscale Scores for Children & Adolescents (N = 50) Subscale Mean Standard Deviation Minimum-Maximum Social Acceptance 3.3 0.7 1.2-4.0 Athletic Competence 3.2 0.7 1.7-4.0 Global Self-Worth 3.5 0.6 1.7-4.0

Since the average grade of the sample was six, the scores from this study were compared to the

subscale means of sixth grade boys assessed by Susan Harter (Harter, 1985a). The means of this

sample are actually higher than the means of the sixth grade sample assessed by Susan Harter as

listed in Appendix G. Independent samples t-test were conducted and the domains of Social

Acceptance (t(67.8) = 2.2, p = 0.03) and Global Self-Worth (t(77.5) = 3.7, p = 0.0004) showed a

significant difference between the Harter reference groups and our sample. There was no

significant difference for Athletic Competence (t(68.17) = 0.2, p = 0.9).

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When looking at the self-perception subscale scores for both children and adolescents, a strong

correlation was found between social participation and Social Acceptance (r = -0.5, p = 0.0003)

as well as between social participation and Global Self-Worth (r = -0.6, p = < 0.0001). The

negative correlation suggests that when children had better self-perception in terms of Social

Acceptance and Global Self-Worth, they tended to report better social participation. The

correlation between social participation and Athletic Competence was smaller and not

statistically significant (r = -0.2, p = 0.1), therefore the null hypothesis cannot be rejected that

there was no correlation between children and adolescents when it came to self-perceived

Athletic Competence and social participation restrictions.

Figure 3. Social Participation and Social Acceptance (Children & Adolescents) (N = 50)

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Figure 3 illustrates the correlation between social participation as measured by the Participation

Scale for Kids, where higher scores indicated more restrictions with participation, and Social

Acceptance as measured by the Self-Perception Profile for Children or Adolescents, where

higher scores indicated more adequate self-perception. The confidence elliptical curve shows

95% prediction.

Figure 4. Social Participation and Global Self-Worth (Children & Adolescents) (N = 50)


Scale for Kids, where higher scores indicated more restrictions with participation, and Global

Self-Worth as measured by the Self-Perception Profile for Children or Adolescents, where higher

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scores indicated more adequate self-perception. The confidence elliptical curve shows 95%

prediction.

Figure 5. Social Participation and Athletic Competence (Children & Adolescents) (N = 50)


Scale for Kids, where higher scores indicated more restrictions with participation, and Athletic

Competence as measured by the Self-Perception Profile for Children or Adolescents, where

higher scores indicated more adequate self-perception. The confidence elliptical curve shows

95% prediction.

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Although there were differences in the correlations found between social participation and self-

perception for children and adolescents, when comparing the means of the children and

adolescent groups, allowing for unequal variances, no significant differences were found for any

of the subscales. Table 7 displays the results of a test that compared the means of children and

adolescents on the self-perception subscales.

Table 7. Comparing the Means of the Self-Perception Subscale Scores for Children &

Adolescents (N = 50) Subscale df t p

Social Acceptance 40.7 -1.4 0.2 Athletic Competence 38.3 -0.2 0.8 Global Self-Worth 42.0 -0.8 0.4

4.7.2.1 Children

The subscale mean scores on the Self-Perception Profile for Children are displayed in Table 8.

Table 8. Self-Perception Subscale Scores for Children (n = 30) Subscale Mean Standard Deviation Minimum-Maximum Social Acceptance 3.2 0.7 1.7-4.0 Athletic Competence 3.2 0.7 1.7-4.0 Global Self-Worth 3.5 0.6 1.7-4.0

The subscale scores for children revealed that there was a strong relationship between social

participation and Social Acceptance (r = -0.6, p = 0.002) as well as between social participation

and Global Self-Worth (r = -0.7, p = < 0.0001). Thus, if a child reported better self-perception

on the subscales of Social Acceptance or Global Self-Worth, he tended to report better social

participation. Again, the correlation between social participation and Athletic Competence was

smaller and not statistically significant (r = -0.1, p = 0.5) and thus the null hypothesis could not

be rejected that there was no correlation between self-perceived Athletic Competence and social

participation for children.

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Figure 6. Social Participation and Social Acceptance (Children) (n = 30)


Scale for Kids, where higher scores indicated more restrictions with participation, and Social

Acceptance as measured by the Self-Perception Profile for Children, where higher scores

indicated more adequate self-perception. The confidence elliptical curve shows 95% prediction.

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Figure 7. Social Participation and Global Self-Worth (Children) (n = 30)


Scale for Kids, where higher scores indicated more restrictions with participation, and Global

Self-Worth as measured by the Self-Perception Profile for Children, where higher scores


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Figure 8. Social Participation and Athletic Competence (Children) (n = 30)



Competence as measured by the Self-Perception Profile for Children, where higher scores


4.7.2.2 Adolescents

The adolescent age group had slightly higher mean scores for Social Acceptance and Global

Self-Worth subscales than the children, however the Athletic Competence subscale mean score

for adolescents was slightly lower than for children. The mean scores for the subscales are

displayed in Table 9.

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Table 9. Self-Perception Subscale Scores for Adolescents (n = 20) Subscale Mean Standard Deviation Minimum-Maximum Social Acceptance 3.5 0.7 1.2-4.0 Athletic Competence 3.2 0.7 2.0-4.0 Global Self-Worth 3.6 0.6 2.2-4.0

Unlike the children, the adolescents had a strong correlation between social participation and

Athletic Competence (r = -0.6, p = 0.005), but the correlations for Social Acceptance (r = -0.3, p

= 0.2) and Global Self-Worth (r = -0.3, p = 0.2) were moderate and not statistically significant.

This result indicates that adolescents who report higher scores in Athletic Competence will also

report that they have better social participation.

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Figure 9. Social Participation and Athletic Competence (Adolescent) (n = 20)



Competence as measured by the Self-Perception Profile for Adolescents, where higher scores



Table 10 displays the subscale scores for the Social Support Scale for Children and Adolescents.

Table 10. Social Support Subscale Scores for Children & Adolescents (N = 50) Children (n = 30)

Subscale Mean Standard Deviation Minimum-Maximum Parental Support/Regard 3.6 0.6 1.8-4.0 Teacher Support/Regard 3.4 0.7 1.2-4.0 Classmate Support/Regard 3.4 0.6 1.3-4.0 Close Friend Support/Regard 3.5 0.5 1.8-4.0

Adolescents (n = 20) Subscale Mean Standard Deviation Minimum-Maximum Parental Support/Regard 3.7 0.4 2.5-4.0 Teacher Support/Regard 3.3 0.8 1.7-4.0 Classmate Support/Regard 3.5 0.6 1.3-4.0 Close Friend Support/Regard 3.5 0.7 1.0-4.0

Appendix H details the correlations for children and adolescents separately. Although

significant correlations were found between social participation and social support for children

only, when comparing the means of the children and adolescent groups, allowing for unequal

variances, no significant differences were found for any of the subscales. Table 11 displays the

results of a test that compared the means of children and adolescents on the social support

subscales. Given that there were no significant differences between the two group means, further

analyses discussed below combined both children and adolescent scores.

Table 11. Comparing the Means of the Social Support Subscale Scores for Children &

Adolescents (N = 50) Subscale df t p

Parental Support/Regard 47.7 -0.3 0.8 Teacher Support/Regard 38.9 0.7 0.5 Classmate Support/Regard 41.2 -0.6 0.6 Close Friend Support/Regard 33.2 -0.1 1.0

Much like self-perception, when the self-reported social support of the sample was compared to

the sample assessed by Susan Harter, it was apparent that the mean subscale scores for sixth

grade boys were higher in the sample of boys with hemophilia (see Appendix I). Indeed, when

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independent samples t-tests were conducted, there were significant differences between the

Harter reference group and our sample in all of the domains (see Appendix J).

A strong correlation was found between social participation and Parental (r = -0.6, p = < 0.0001),

Teacher (r = -0.5, p = < 0.0001), and Classmate (r = -0.6, p = < 0.0001) Support/Regard.

Moderate correlations were found between social participation and Close Friend (r = -0.4, p =

0.002) Support/Regard. These results indicated that children who endorsed better support or

regard from their parents, classmates, teachers, and close friends also endorsed that they have

better social participation.

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Figure 10. Social Participation and Parental Support/Regard (N = 50)


Scale for Kids, where higher scores indicated more restrictions with participation, and Parental

Support/Regard as measured by the Social Support Scale for Children, where higher scores

indicated more perceived social support. The confidence elliptical curve shows 95% prediction.

Figure 11. Social Participation and Teacher Support/Regard (N = 50).


Scale for Kids, where higher scores indicated more restrictions with participation, and Teacher

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Figure 12. Social Participation and Classmate Support/Regard (N = 50)


Scale for Kids, where higher scores indicated more restrictions with participation, and Classmate



90

Figure 13. Social Participation and Close Friend Support/Regard (N = 50)


Scale for Kids, where higher scores indicated more restrictions with participation, and Close

Friend Support/Regard as measured by the Social Support Scale for Children, where higher

scores indicated more perceived social support. The confidence elliptical curve shows 95%

prediction.

When it came to disclosing their hemophilia to others, the participants in this sample tended to

disclose to most people in their life. Of the 50 participants, 47 indicated that their family

members knew (94.0%), 31 had told their close or best friends (62.0%), 35 had told their

teachers (70.0%), and 24 had told their classmates (48.0%).

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4.7.4 Family Impact

When it came to the impact on family members, the scores on the PedsQL™ Family Impact

Module were high, which suggested that overall the participants had a low negative impact on

the family.

There was a moderate correlation between social participation and the subscales tapping family’s

Social Functioning, which indicated that when parents/guardians reported better social

functioning, their children tended to have better social participation.

All other subscales did not have statistically significant correlations. The results of the PedsQL™

Family Impact Module are in Table 12.

Table 12. PedsQL™ Family Impact Module Results and Correlation with the Participation Scale

for Kids (N = 50) Variable Mean Standard Deviation Minimum-Maximum Pearson r Total Score 3074.0 571.1 1275.0-3600.0 -0.2 Physical Functioning 91.4 14.0 37.5-100.0 -0.2 Emotional Functioning 80.8 19.7 25.0-100.0 -0.2 Social Functioning 89.8 18.9 25.0-100.0 -0.3* Cognitive Functioning 90.5 19.9 20.0-100.0 -0.2 Communication 80.8 20.2 25.0-100.0 -0.2 Worry 73.2 22.5 15.0-100.0 0.0 Daily Activities 88.8 19.1 33.3-100.0 -0.3 Family Relationships 87.9 18.1 20.0-100.0 -0.2 Note. *Correlation is significant at p < 0.05.

4.8 Model Checking

For each Pearson correlation coefficient, a number of tests were run to verify that data

assumptions were held.

4.8.1 Normality of Residuals

Residuals were considered normally distributed if the Shapiro-Wilk test was non-significant.

The normality of residuals are listed in Appendix K. Nearly all the Shapiro-Wilk tests were

significant (except for Athletic Competence for Adolescents and Parental Support/Regard), thus

the data do not meet the assumption of homoscedasticity. However, the central limit theorem

states that given a large enough sample size, the means of the data will be normally distributed,

even if the original distribution is not. Therefore, since the data is being used to make inferences

on means, the use of parametric statistics such as Pearson correlations is justified.

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4.8.2 Influential Outliers

Observations were identified as influential outliers if the DFFIT value was greater than 2, a

common rule of thumb (Vogt & Johnson, 2011). No influential outliers were identified using

this test.

4.8.3 Straight-Line Relationship and Homoscedasticity

Residual plots constructed for the relationship between social participation and self-perception

(Social Acceptance, Athletic Competence, and Global Self-Worth subscales), social support

(Parental, Teacher, Classmate, Close Friend Support/Regard subscales), and Impact on the

Family (Physical Functioning, Emotional Functioning, Social Functioning, Cognitive

Functioning, Communication, Worry, Daily Activities, and Family Relationships subscales)

identified some relationships and patterns, suggesting that perhaps the residuals were not

normally distributed. Residual plots are displayed in Appendix L.

4.8.4 Non-Parametric Tests

Non-parametric tests were conducted for comparison purposes given that some assumptions for

the use of Pearson correlations were violated. Spearman’s rank-order correlations were

compared to Pearson correlation coefficients and some changes were observed regarding the

strength of the correlation, however the outcomes were essentially unchanged. See Appendix M

for a detailed analysis.

The fact that the residuals are non-normal suggests that there may be some deviations from the

linear model. These changes may be partially due to the fact that the data collected was ordinal

in nature rather than interval or ratio. Ordinal data is often assessed using parametric tests and

given that the results of the non-parametric tests were not substantially different from those of

the parametric tests, we have chosen to report the Pearson correlation coefficients.

4.9 Sensitivity Analysis

To test the robustness of this analysis, sensitivity analysis of a worst- and best-case scenario were

performed for missing data. Minimum values were specified as the 10th percentile, while

maximum values were specified as the 90th percentile. In keeping with the previously reported

data, Pearson correlation coefficients were performed on the data.

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Since less than 5% of the data was missing for any one variable, there were minimal changes

witnessed in the re-analysis. Only data that reached statistical significance with single

imputation were discussed. Data are presented in Appendix N.

4.9.1 Worst-Case Scenario

When imputing the 10th percentile for missing values, correlations were essentially unchanged

for all three of the self-perception subscales for children and adolescents, children alone, and

adolescents alone.

Nearly imperceptible changes were observed between social participation and social support for

all four subscales.

A very minor change was seen between social participation and impact on the family. Social

Functioning no longer met significance (r = -0.3, p = 0.1) while Daily Functioning met

significance (r = -0.3, p = 0.05). Again, these changes were very minor and the correlations

moderate at best.

4.9.2 Best-Case Scenario

Using the 90th percentile for missing values yielded fewer changes to the analysis. Social

Functioning no longer met significance (r = -0.3, p = 0.1).

4.10 Linear Modeling

Multiple linear regressions were constructed to examine if social participation was affected by

self-perception and social support. Although the ICF model suggests that the relationships

between all domains are bidirectional, the data seemed appropriate for exploratory analysis to

determine if any of the variance in social participation could be accounted for by these variables.


When looking at social participation and self-perception for all participants, the omnibus F-test

was significant and thus individual covariates were further evaluated for significance, as shown

in Table 13.

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Table 13. Parameter Estimates for Linear Regression of Social Participation and Self-

Perception Predictor Parameter Estimate Standard Error Test Statistic p-value Omnibus F-test (F(ndf,ddf)) 10.4 (3, 46) < 0.0001 Social Acceptance -1.4 1.3 -1.1 0.3 Athletic Competence -0.6 1.1 -0.6 0.6 Global Self-Worth -5.3 1.5 -3.5 0.001 Note. R2 = 0.4

This model suggested that 40.2% of the variance in social participation could have been

explained by self-perception; in particular it was Global Self-Worth that exerted an effect.


The use of linear modeling requires that the data meet several assumptions. The data for self-

perception and social participation did not meet all the required assumptions; therefore a linear

model may not be best suited for this data.

4.10.1.1.1 Normality of Residuals

Residuals initially appeared to be normally distributed when using graphical tools (histograms

and Q-Q plots, see Appendix O) and were not skewed (skewness = 1.0). However, when model

assumptions of normality were determined using the Shapiro-Wilk test, the significant test result

(W = 0.9, p = 0.01) suggested that the residuals were in fact from a non-normally distributed

population. Since the residuals were not normally distributed, there may have been a non-linear

relationship between social participation and self-perception and linear regression may not have

been the best fit.

4.10.1.1.2 Influential Outliers

No influential outliers were identified when using tests of DFFIT.

4.10.1.1.3 Straight-Line Relationship and Homoscedasticity

To verify the straight-line relationship and homoscedasticity, residual plots were created. There

was no obvious relationship or pattern visible from the residual plot. However when variation

around the predicted line was plotted, the residuals did not follow a straight line nor did they

appear to be homoscedastic, suggesting that a linear line may not have been appropriate for the

data.

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Tests for collinearity used a Variance Inflation Factor cut-off of > 4.00 to indicate collinearity

(Victor, 2012). The tests showed that Social Acceptance (VIF = 1.8), Athletic Competence (VIF

= 1.2), Global Self-Worth (VIF = 1.6) were not collinear with one another.


The omnibus F-test for social participation and social support was significant and therefore

individual covariates were further examined as shown in Table 14.

Table 14. Parameter Estimates for Linear Regression of Social Participation and Social Support Predictor Parameter Estimate Standard Error Test Statistic p-value Omnibus F-test (F(ndf,ddf)) 11.5 (4, 45) < 0.0001 Parental Support/Regard -4.2 1.9 -2.2 0.0 Teacher Support/Regard -1.1 1.3 -0.8 0.4 Classmate Support/Regard -4.2 1.8 -2.3 0.0 Close Friend Support/Regard 0.6 1.7 0.4 0.7 Note. R2 = 0.5.

According to this data, social support explains 50.6% of the variance in social participation and

the domains of parental and classmate support/regard are the domains that have the strongest

influence.


Again, the data for social support and social participation did not meet the assumptions required

for the use of linear modeling. Linear models may not best describe the data presented.

4.10.2.1.1 Normality of Residuals

Graphical tools such as histograms and a Q-Q plot initially suggested that the residuals were

relatively normal (see Appendix P). However, the Shapiro-Wilk test used to determine

normality was significant and therefore the residuals were not normally distributed (W = 1.0, p =

0.05). Residuals were skewed (0.6) but within an acceptable range; kurtosis was also acceptable

(1.2).

4.10.2.1.2 Influential Outliers

No influential outliers were identified when using tests of DFFIT.

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4.10.2.1.3 Straight-Line Relationship and Homoscedasticity

Residual plots again did not indicate any relationships however variation around the predicted

line showed a non-linear relationship and lack of homoscedasticity.

Again, tests for collinearity using a cut-off of > 4.0 on the Variance Inflation Factor were used.

The results showed that none of the domains were collinear (Parental VIF = 2.3, Teacher VIF =

2.3, Classmate VIF = 2.7, Close Friend VIF = 2.5).

4.10.3 Stratification Variables

Multiple linear regressions were conducted using age and factor level as predictor variables of

social participation and self-perception. None of the models achieved significance. See

Appendix Q.

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Chapter 5 Discussion

5 Interpretation of Findings Hemophilia is a condition that can influence many factors in one’s life. The ICF model suggests

that there is a dynamic interaction between hemophilia and the personal and environmental

factors in an individual’s life. These factors are engaged in a bilateral relationship, where

changes in one factor can lead to changes in the other factors. Using this model, the data support

the conclusion that the personal factor self-perception contributes to his social participation.

Similarly, social support is an environmental factor that can impact social participation. Given

the bilateral nature of the model, social participation will also exert an influence on these

contextual factors as well. It is the interplay observed between all of these variables that

determines one’s functioning and disability, including one’s social participation.

The results of this study indicated that boys with hemophilia in Toronto, Ontario, had minimal

social participation restrictions, high levels of perceived competence, and perceived that other

supported them. This study was highly generalizable given the sampling strategy. There appears

to have been moderate to strong relationships between social participation and self-perception as

well as social participation and social support. There was little relationship observed between

social participation and impact on the family.

5.1 Sample

There was a high participation rate in this study with very few patients declining to participate.

Research staff was cognizant of the burden of participating in the research study and thus

structured the study visit to coincide with a regularly scheduled clinic appointment. This ensured

that extra trips to the hospital were not required. Research staff was also aware that participants

may fatigue throughout the multiple assessments and encouraged participants and their family

members to take breaks as needed. By offering to remove as many barriers to participation as

possible, research staff was able to increase recruitment.

The participants came largely from married or common-law households and identified both their

mother and father as their primary guardians. In spite of the extreme stressors and pressures of

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illness on the family, the findings of this study support the works of others who have found a

high rate of marriage amongst parents of children with hemophilia and other illnesses (Cadman,

Rosenbaum, Boyle, & Offord, 1991; Kellerman et al., 1980; Varekamp et al., 1990; Wiedebusch,

Pollmann, Siegmund, & Muthny, 2008).

Treatment for the majority of participants in the study cohort happened in the home with

infusions typically being administered by oneself or by one’s mother. Other studies have also

found that mothers tended to be more involved in the daily care of the patient (Beeton et al.,

2007) and that fathers were less likely to infuse (Herrick, Nussbaum, Holtzman, & Wissow,

2004). Possible reasons are that mothers may be more likely to stay at home with an ill child

(Lindvall et al., 2012) and therefore more likely to attend doctors appointments. The mother

may feel more comfortable administering the treatment initially due to increased exposure and

this may continue to perpetuate with the mother administering treatment more often.

Furthermore, mothers in particular have been identified as being overprotective of the child with

hemophilia, perhaps due to feelings of guilt about the genetic transmission of the disease (Agle,

1984).

Attending school increases the opportunities for social participation. Nearly all of the

participants in this sample attended school, which was in keeping with research completed in the

United States that found approximately 98% of their sample of boys with hemophilia attended

school and 2% were home-schooled (Shapiro et al., 2001). Shapiro et al. (2001) found a

significant difference in school attendance between participants who had a higher number of

bleeding episodes (defined as 12 or more bleeding episodes in the year before enrollment) than

the participants who had fewer bleeding episodes. Although there may be differences in school

attendance within the hemophilia population, research conducted as early as 1987 in Finland

between persons with hemophilia and controls found no significant differences in the number of

school hours absent (B. Kvist et al., 1990).

School attendance today is in sharp contrast to data from 1971, which found that persons with

hemophilia were absent from school more than double the hours of controls (B. Kvist et al.,

1990). The increased school attendance may be attributed to advances in treatment. This sample

was composed of participants primarily treated with prophylactic therapy, which essentially

converts a severe to a moderate phenotype (Feldman, Pai, Rivard, & Israels, 2006). Having

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access to treatment and a prophylactic treatment regimen can minimize bleeds, allowing the

person to participate more and engage in regular activities including school.

5.2 Social Participation

Participation was defined as “involvement in a life situation” (World Health Organization, 2001)

and it was important that the measure used took into account an assessment of the subject’s

limitations or restrictions against the norm. The measure chosen included an element of

desirability in its measurement of social participation. Although much of the research conducted

to date on participation has focused on sport, the goal of this study was to take a more inclusive

approach to participation and assess all aspects of social life. It is evident that the concept of

participation encompasses many facets in the respondents’ minds. The Participation Scale for

Kids did not have an explicit focus on sport, which allowed for participants not interested in

sport to still express their ability to participate.

An important finding of this study was that most of the sample reported that they experienced

very few social participation restrictions. This may be due to the fact that the majority of the

sample was on prophylaxis, allowing the study subject to engage in activities similar to their

peers. This is comparable to the Western European sample studied by Gringeri et al. (2004),

who found that patients on prophylaxis were less impaired than those using on-demand

treatment. Similar to Khair et al.’s (2011) United Kingdom sample, most of this sample felt that

their hemophilia did not prevent them from participating like their peers. As discussed earlier,

samples that were studied in the 1970s prior to the advent of prophylaxis would have had

markedly different social participation as patients may not have been able to attend school

regularly and may have suffered from hemophilic arthropathy at an early age.

When this sample was asked specifically if their hemophilia prevented them from participating

like their peers, the majority of positive answers related to inability to participate in certain

sports. Since sport is desirable in Western culture (Heijnen et al., 2000), it was not a surprise

that many of the boys wished to be able to participate similar to their peers. More importantly,

early adolescence is a time to develop a sense of industry or mastery of skills, in physical,

academic, and social tasks (Manning, 1988). Spending time in activities that help the child build

his competencies may be critically important in shaping the child’s development (Mahoney,

2005, as cited in Slutzky & Simpkins, 2009). Being able to competently engage in sport may be

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especially salient for boys, as boys tend to spend more time in sports and report higher sport self-

concepts and stronger beliefs in the value of sports than girls (Slutzky & Simpkins, 2009).

Denying children the opportunity to engage in sport as their peers may thwart opportunities to

gain competence in that area and could lead to lower self-perception. Slutzky and Simpkins

(2009) found that the more time children participated in team sports, the better they felt about

their athletic competency, which was positively associated with self-esteem. Sport participation

for children with low peer acceptance may be particularly beneficial as sport has the potential to

address both peer relationships and individual development (Smith, 2003; Weiss & Duncan,

1992, as cited in Slutzky & Simpkins, 2009).

Only a small number of participants indicated that they experienced mild or moderate

participation restrictions. Significantly, not one participant endorsed experiencing severe

participation restrictions. This finding is consistent with study results from Heijnen et al. (2000),

who found that 95% of boys with hemophilia in their study in the Netherlands were able to

participate in physical education classes. It appears that although the boys may be prevented

from participating in certain sports, they were still able to socially participate on par with their

peers. In fact, many boys with hemophilia actively engage in sports such as swimming, cycling,

tennis, skating, and skiing (Heijnen et al., 2000).

Children had a slightly higher number of participation restrictions than adolescents due to a

couple of influential outliers who had mild to moderate participation limitations. These four

participants skewed the mean and they were different than the rest of the sample because the

majority of the sample indicated that they experienced no participation restrictions. These four

participants were also some of the youngest participants in the sample, and it may be that these

participants felt more limited or overprotected by their parents/guardians or teachers. Hamburg

(1983, as cited in Spirito et al., 1990) noted that the risk of parental overprotection was

particularly striking for children with chronic illnesses and that this could lead to limited

opportunities for socialization. Well-meaning friends and family members may hamper the

child’s ability to fend for himself and make it difficult for the patient to feel a sense of

empowerment and capability (Thomas & Gaslin, 2001). While taking inappropriate risks are not

encouraged, avoiding any activity with the potential for injury would lead to a sheltered life that

would not allow for adequate development of the self and could lead to feelings of inadequacy

and inferiority (Manning, 1988).

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Adolescents reported fewer social participation restrictions than the children in this sample and

all adolescent scores fell below the mild restriction range. This data suggests that as children

age, the number of restrictions to their social participation decreases. It may be that as children

get older, they adapt their activities to be in line with things that they have fewer participation

restrictions with such as less physically strenuous activities or more academically inclined

activities. Research conducted by Köiter et al. (2009) found that the choice of sports that

patients with hemophilia engaged in appeared to be based on adaptation to their disease. Heijnen

et al. (2000) had similar findings as they concluded that most boys and men with hemophilia

were sensible in choosing a sport that accommodated their hemophilia, as many indicated they

would like to play soccer but did not because of their disease. Our sample echoed these thoughts

as those who commented that their hemophilia restricted them identified contact sports such as

American football and ice hockey as being off-limits. As stated earlier, however, most of the

participants felt that they did not experience social participation restriction due to their

hemophilia.

Another possibility as to why adolescents did not experience as many social participation

restrictions as children is that adolescents may engage in activities that their parents forbid

(Buzzard, 1996). Thus the adolescent participants may have reported fewer participation

restrictions because they were able to engage in those activities that they enjoyed, regardless of

whether or not their parents supported it. This arises in part because children begin to conform

to their peers as they progress through their elementary school years (Spirito et al., 1990). Peers

become the benchmark upon which the child measures his success and peers’ opinion of their

success or failure may be of far more importance to the child than the opinions of his parents or

teachers (Maier, 1969, as cited in Manning, 1988). Adolescents are also beginning to search for

their identity at this age and may be concerned about how they appear to others as compared to

how they feel they are (Manning, 1988).

As the Participation Scale for Kids scale was modified and administered for the first time to this

sample, there was no reference group for comparison. It is also possible that some of the

younger participants did not understand the open-ended questions at the end of the instrument

and it may have been more appropriate to explore these topics with the older participants instead.

However, the internal consistency of the scale was good and the results of the correlations

between social participation, self-perception, and social support suggest good convergent

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construct validity. Given the very few identified participation restrictions, it can be concluded

that children with hemophilia in Toronto, Canada, seem to have relatively few social

participation limitations. Difficulties that arise with social participation may impact and be

impacted by factors such as self-perception and social support, as well as issues with body

function and structure.

It is important to remember that although the majority of participants with hemophilia in this

sample were able to participate on par with their peers, there were some participants who

reported very low self-perception scores in each of the domains. In spite of all that has been

achieved, it is important to bear in mind that chronic illness is a part of these boys’ lives and

sometimes it can be a very difficult illness to cope with (Thomas & Gaslin, 2001).

5.3 Self-Perception

Self-perception is defined as one’s perception of themselves, which may include his or her

feelings of competence and their overall self-worth (Harter, 1985a). Despite living with a

chronic illness, self-perception in this sample was actually higher than the reference groups

studied by Harter et al. (1985) in Colorado, United States. Previous research has not come to a

unanimous conclusion about self-perception in boys with hemophilia. Some have suggested that

boys with hemophilia are more likely to have lower feelings of perceived competence (Agle,

1984), while others have said that self-esteem and self-perception are mostly comparable to

healthy peers (Hegeman et al., 2011; Kellerman et al., 1980). Regardless, Stein and Jessop

(1984, as cited in Thomas & Gaslin, 2001) note that self-esteem deteriorates and health status

subsequently worsens if children are unable to perform age-appropriate roles and tasks.

Global Self-Worth in this sample was the perceived competence that had the highest rating. If a

child indicated higher scores on the Global Self-Worth subscale, this meant that the child liked

themselves as a person, were happy with the way they were leading their life, and were generally

happy with the way they were (Harter, 1985a). Social Acceptance was the next highest scoring

subscale and this suggested that the boys with hemophilia generally felt accepted by their peers

and/or felt popular. The lowest scoring subscale was Athletic Competence, which examined the

boys’ sports and outdoor games competency, for which most boys felt adequate. The reference

scores for sixth grade boys (Samples A and B) were similarly ordered with the highest subscale

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scores in the domain of Global Self-Worth, followed by Athletic Competence, and then Social

Acceptance (Harter, 1985a).

The higher mean score in this sample may be due to the fact that this sample had a wider range

of grades completing the survey (participants ranged in grade from grades 2-12) and older

participants tended to report higher scores on the self-perception subscales than the younger

participants. There may be more variability of scores within one grade (reference scores were

based on sixth grade boys) than across ten grades (this sample). Furthermore, the reference

scores were gathered thirty years earlier (1980s), drew on different neighborhoods (lower middle

class to upper middle class), was mostly Caucasian (approximately 90% of the sample), and from

a different country (United States). All these factors may have contributed to differing mean

scores.

Alternatively, it may be that these findings support the notion that children with hemophilia are

no more susceptible to lower levels of self-perception than their peers. This is in line with recent

research by Hegeman et al. (2011) who found that in general, self-perception scores were

comparable between children with hemophilia and healthy peers. Older research that compared

multiple illnesses (oncology, cardiology, diabetes, cystic fibrosis, nephrology, and

rheumatology) and healthy controls also found no differences in self-esteem between the various

illness groups or between the healthy controls and the ill groups (Kellerman et al., 1980).

Hemophilia shares some similarities with diabetes, as both illnesses allow for patients to learn

self-injection/infusion techniques. Kellerman et al.’s (1980) study also looked at locus of

control, which the authors defined as one’s attitude towards his ability to control his own destiny.

They found that there was no difference in locus of control between the healthy group and the

diabetes or cystic fibrosis groups. The nature of chronic illness “often opposes children’s

developmental striving for independence” (Coupey and Cohen, 1984, as cited in Thomas &

Gaslin, 2001), but mastering the task of self-injection/infusion may allow for the patient to feel

that he is more in control of his future and feelings of industry can contribute to greater self-

perception.

While earlier research theorized difficulties for boys with hemophilia, including low self-esteem

(Agle, 1984), self-perception was found to be quite high for our sample. The difficulties that

boys with hemophilia faced in the 1980s may be quite different than the challenges experienced

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today. Patients today have access to ultra-safe, effective therapy and prophylactic regimens that

allowed for safe engagement in nearly all activities. Participating in activities like one’s peers

may allow the boy with hemophilia to not only develop a sense of mastery, but also be accepted

by his peers. Both of those may contribute to feelings of competence and self-perception and

vice versa.

Alternatively, it is possible that this sample was less likely to endorse feelings of negative self-

worth in the presence of their medical team or their family members, who were often present

when study questionnaires were being completed. Participants may not have wanted to worry

their medical team or family members and thus endorsed more positive feelings of self-worth.

That being said, steps were taken to reduce social desirability, as participants were encouraged to

complete the questionnaires in a self-report manner whenever possible and individual

questionnaire results were not shared with their medical team. Furthermore, the setting in which

this research was conducted was likely similar to other research carried out in hospital clinics.

The results of this study are consistent with those reported by Trzepacz et al. (2003), who found

differences between boys with hemophilia and comparison peers in the United States on the

dimensions of Scholastic Competence, Athletic Competence, and Global Self-Worth, but the

scores were still within the normal range. Hegeman et al. (2011) in the Netherlands found that

for the most part, boys with hemophilia had perceived competence on par with a normative

sample. The only significant differences found were in the domains of Global Self-Worth for

children and Close Friendship for adolescents but these scores were still relatively high,

indicating better self-perception. The results of this sample support the findings of other studies

in developed countries and clearly demonstrate that boys with hemophilia treated with

prophylaxis did not have reduced levels of self-perception.

5.3.1 Primary Hypothesis

The primary study hypothesis was that an association existed between self-perception and social

participation. The present data shows that overall there were moderate to strong correlations

between social participation and the three self-perception subscales used in this study. Thus,

participants who reported more adequate self-perception in terms of Social Acceptance, Athletic

Competence, and Global Self-Worth also reported fewer social participation restrictions.

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Accordingly, those participants who experienced more difficulties with social participation had

lower scores on self-perception subscales.

Social Acceptance was moderately associated with social participation for younger boys with

hemophilia, which shows that peer integration may be related to social participation. The

associations between social participation and Social Acceptance were lower and not statistically

significant for adolescents, which would seem to indicate that participating socially and having a

sense of being accepted by one’s peers may be less related for adolescents than for children.

This result was unexpected because it was initially hypothesized that adolescents who felt

competent in the domain of Social Acceptance would also experience fewer restrictions in social

participation. It may be that our sample of adolescents was too small (n = 20) to find a

significant effect size, or it may be that as adolescents aged, they were faced with more

participation restrictions compared to their peers but continued to have perceived competence in

the area of Social Acceptance.

Indeed, Heijnen et al. (2000) found that 68.0% of their sample of older boys with hemophilia in

the Netherlands reported that they sometimes did not attend physical education sessions because

of bleedings, a clear restriction on their social participation. Other work in the Netherlands by

Hegeman et al. (2011) found that adolescents with hemophilia reported high levels of Social

Acceptance, albeit with scores lower than the Dutch reference group. Our data appear to reflect

the work carried out in the Netherlands and show that even when faced with social participation

restrictions, one can have good self-perception in the domain of Social Acceptance.

There was a lack of correlation between social participation and Athletic Competence when

children and adolescents were combined in the analysis. There was a significant correlation

when only adolescents were examined, which would seem to indicate that sports and outdoor

games are more intimately tied to social participation for adolescents than children. For

adolescents, Athletic Competence was the most strongly associated subscale with social

participation, suggesting that as children get older Athletic Competence becomes more strongly

associated with social participation. This finding is not surprising as older children may become

more involved in team sports and gain competence and mastery of the skills required to

participate in sports and outdoor games, which could then significantly affect one’s social

participation.

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As described earlier, sporting activity was the participation restriction cited most often by this

sample. In line with previous recommendations (Buzzard, 1996), participants noted that their

hemophilia tended to prevent them from participating in contact sports. Our data show that some

children with hemophilia had very high levels of athletic competence but some had very low

levels. It is possible that since Western culture desires sport participation (Heijnen et al., 2000),

participation in sport as a child is expected and accepted as part of daily life. Those who do not

thrive in sport may self-select themselves out of sport-related activities as they get older but

participate fully in other activities. Thus, one may find children with high levels of social

participation and low levels of Athletic Competence and vice versa. Alternatively, younger

children in the sample may be less engaged in organized sport than their adolescent counterparts

and therefore Athletic Competence may still be in the developing stages for some participants.

The Global Self-Worth subscale showed differences in the sample between the children and

adolescents. For children, social participation was most strongly associated with the subscale of

Global Self-Worth, indicating that when children feel generally happy with themselves as a

person, they also tend to engage in social participation more. On the other hand, the data for

adolescents was not sufficient to reject the null hypothesis that there was no association between

social participation and Global Self-Worth. As discussed above, it appears that as children get

older, other aspects (such as Athletic Competence) become more salient and more associated

with social participation than Global Self-Worth and this may partially explain the differences

observed.

It has been suggested that children with strong self-esteem are better able to handle challenges of

daily life (White, 1998, as cited in Hegeman et al., 2011; Thomas & Gaslin, 2001). Thus,

developing strong self-esteem and self-worth may be crucial to an individual’s success and social

participation, especially in the case of children with hemophilia where some may be vulnerable

to lower self-worth.

5.4 Social Support

Overall, boys in this study felt that they were well supported by their parents, teachers,

classmates, and close friends. This is in contrast to previous research that has found that

adolescents and adults with hemophilia report difficulties with perceived support (Gringeri et al.,

2004; Iannone et al., 2012). One reason for the differences observed may be that this sample was

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predominantly composed of children, who may be exposed to a wider support network than

adults.

The importance of a social support network has far-reaching consequences. If a patient

perceives that he has a good social support network, he may feel less depressed, have better

compliance, and higher health-related quality of life (Gringeri et al., 2004; Iannone et al., 2012;

Petrini & Seuser, 2009). Developing a strong social support network for the patient may prevent

patients from concealing dangerous or risk-taking behaviors. Buzzard (1996) notes that if a

patient is forbidden from participating in certain sports, “there is a good chance that he…will

play it anyway without the parents’ knowledge.”

There is evidence that perceived emotional social support may be protective in the psychological

adjustment of children with cancer and their siblings. In the United States, it was found that

classmate social support was the most important factor in the psychological adjustment of newly

diagnosed cancer patients (Varni, Katz, Colegrove, & Dolgin, 1994). Meanwhile it was found

that Canadian siblings with stronger perceived social support reported fewer depressive and

anxiety symptoms, and their parents reported fewer behavioral problems (Barrera, Fleming, &

Khan, 2003). Children who have good social support may also have better psychological

adjustment, as they would have resolved the identity versus role confusion stage and developed a

strong sense of self. This may, in turn, increase the likelihood that they have higher levels of

social participation.

As children progress through the normal developmental stages, they begin to carve out identities

for themselves and may adopt idols and heroes to whom they look up to. Parents and teachers

may find themselves in these roles and may need to provide support to the child as he discovers

inevitably that his ideological thinking is not realistic. Parents and teachers may also need to

adjust to the changing identities of the child and they can nurture these relationships to help the

adolescent pass through the identity versus role confusion stage.

Peers can also have a substantial impact on the adolescent’s search for identity. Erikson (1963,

as cited in Manning, 1988) noted that cliques and crowds can be “remarkably clannish and cruel

in their exclusion of those who are different.” The boy with hemophilia may already feel

different than his peers and has to overcome that hurdle in addition to the normal adolescents

worries and concerns. Although there is little that can be done to abolish cliques and clans from

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the schoolyard, having the support of just one close friend or classmate may be enough for the

child to feel he is cared for and loved.

One difference between the reference groups and this sample is that in the reference groups,

parent and teacher support was systematically higher than support from classmates and close

friends (Harter, 1985a). Our sample found the highest levels of perceived support to come from

parents, close friends, and classmates. Teacher support was lower overall, but the scores were

still quite high. Harter (1985b) suggests that teacher support tends to decline as a function of

grade level. Given that our sample had a wider grade range (participants in the study were in

grades 2-12), the larger proportion of older participants may have had an influence on teacher

support overall. Indeed, our sample indicates that younger participants felt more supported by

their teachers than the older participants, which may have lowered the overall mean score in the

study cohort.

Participants in our sample tended to disclose their illness to others, although not at the same rate

as in other studies. Less than two-thirds of the sample had disclosed their illness to their close or

best friends, in contrast to the 94.0% who had disclosed their illness to friends in a study by

Lindvall et al. (2006). Participants were not probed as to why they had not disclosed their illness

to others, but perhaps since the participants in this sample encountered relatively few

participation restrictions, they felt it unnecessary to disclose their illness to others.

5.4.1 Secondary Hypothesis

Regarding the second aim of this study, results indicate that social support and social

participation were associated. There was a moderate correlation between social participation and

support from close friends, but a strong correlation between social participation and support from

classmates. Since close friends at this age are also likely one’s classmates, it is not surprising

that the correlation is higher for classmates. In fact it is not uncommon for classmates to be used

as a peer control group when studying children (e.g. B. Kvist et al., 1990; Noll, LeRoy,

Bukowski, Rogosch, & Kulkarni, 1991; Trzepacz et al., 2003). Participants with close friends

and classmates whom they feel supported by will thus have peers with whom they can socially

participate alongside.

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A strong correlation was found between social participation and support from parents. Strong

perceived social support from one’s parents might enable a child to participate more readily if

given permission or encouraged to participate in activities. Indeed it has been documented that

parents may struggle with wanting their child to have a normal active lifestyle but also wishing

to protect the child from risk, leading to restrictions on the child’s participation (Shaw & Riley,

2008). However, as discussed earlier, not having the support of one’s parents may not be

sufficient to prevent children from participating in those restricted activities. Having parental

support may include open communication between the patient and the parent about activities he

wishes to participate in. A compromise may be possible that allows the patient to engage in said

activity safely, such as using protective gear or modifying prophylaxis appropriately. Although

the parent may ultimately object to the patient’s participation in said activity, the patient may still

benefit from feeling that his opinion was important, another aspect of social participation.

A modest correlation was found between social participation and support from teachers. Support

from one’s teacher may be especially important in physical education classes. Knowledge of the

patient’s strengths and limitations would be an asset to encourage appropriate levels of

participation (Heijnen et al., 2000). Furthermore, there has been some evidence that patients

with hemophilia may be subject to bullying (B. Kvist et al., 1990) and having a teacher that one

can open up to about such experiences may make the child feel safer at school and allow for

appropriate intervention, if necessary.

Manning (1988) described the implications for educational practice when it came to assisting

students through the early adolescent stages. The author concluded that the significant adults in

the student’s life would greatly impact whether a young person emerged from adolescence with

feelings of industry and the development of a strong personal identity. Without support from

adults whom they can trust, children may feel inferior and confused about demonstrating roles,

and this may hamper their pursuit of social participation.

5.5 Impact on the Family

According to the present data, hemophilia had a relatively limited impact on the family with few

parents or guardians reporting any difficulties. Parents reported the most difficulties in the

domain of Worry, but the mean score for Worry was still high, indicating good family

functioning. This finding is not surprising as Wiedebusch et al. (2008) found parents of children

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with hemophilia reported fewer psychosocial strains when compared to parents of children with

diabetes and rheumatologic diseases.

An earlier study in the Netherlands found that activities or relations were almost never disturbed

because of extra care required for patients with hemophilia; hemophilia was perceived as a minor

burden on daily practical problems (Varekamp et al., 1990). More recently, an international

multi-center cohort study in Malmö, Stockholm, London, and Toronto found that caregivers of

boys with hemophilia and inhibitors reported the disease was a medium burden, while parents of

children without inhibitors reported a low burden (Lindvall et al., 2012). There was also a

significant difference in scores between those with inhibitors and those without on the Impact on

Family Scale, with caregivers of children with inhibitors reporting more negative impact on the

family. Another earlier study in the United States found that approximately one-fifth of parents

had difficulty adjusting to hemophilia, but it is important to note that approximately one-fifth of

that sample also had HIV co-infection, which could have drastically changed the patient’s health

trajectory (Drotar et al., 1997). However it appears that in this sample, where children with co-

morbidities were excluded and none of the participants had inhibitors at the time of the study,

overall family functioning was good.

Some authors have noted that the initial diagnosis can be very stressful for the family of a child

with hemophilia (Agle, 1984; Coppola et al., 2011), especially if there is no family history of

hemophilia. It is possible that the time of diagnosis may be the most stressful event that a family

has yet to encounter. However, given that the minimum age for participation in this study was 7

years old, most of the participants in the study had been diagnosed for several years prior to

participation in the study. It may be that parents and participants alike had habituated to the

illness and thus the illness was seen to have relatively little impact on the family in the four

weeks prior to the assessment. Furthermore, this study was a subset of a larger study and one of

the criteria for inclusion was that participants could not have had an acute bleed within two

weeks of the clinical assessment. Thus we were studying participants when they may have been

at their best and consequently the illness would have had minimal impact on the family in the

previous four weeks.

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5.5.1 Tertiary Hypothesis

When it came to the third hypothesis that impact on the family and social participation were

associated, the data mostly did not support this hypothesis and thus the null hypothesis that there

was no association cannot be rejected. Only modest correlations were found between social

participation and Social Functioning and Daily Activities. These correlations show that

participants who reported better social participation tended to have parents who felt they had

better functioning in social and daily activities. If parents felt that they had a good social

network and did not feel greatly distressed by daily activities, it stands to reason that they would

encourage their children to expand their social activities as well.

5.6 Exploratory Analyses

Recognizing that the ICF model suggests that there is a bidirectional relationship between

personal factors and social participation, linear modeling was used to explore the possibility that

personal and environmental factors may exert influence on one’s social participation.


The linear model strengthens the finding of moderate associations between social participation

and self-perception, as 40.2% of the variance in social participation was explained by self-

perception in this model. Specifically, it was the competence of Global Self-Worth that exerted

the most effect. Given this data, the conclusion was that if a participant exhibited more adequate

Global Self-Worth, he would then perceive fewer social participation restrictions. This finding

was supported by the work of Katz (1970, as cited in Agle, 1975), who noted that if a patient had

an image of being disabled and inadequate, he may have had an inability to assume social and

vocational responsibilities as an adult.


When it came to social support, the linear model found that 50.6% of the variance in social

participation could be explained by social support. The domains of parental and classmate

support/regard had the strongest effects on this model. This finding lead to the conclusion that if

a boy with hemophilia had good support from parents and classmates, he would be more likely to

engage in social participation. Other studies have found that with adequate social support, levels

of depression are lower (Iannone et al., 2012) and quality of life increases (Bullinger, Von

112

Mackensen, Haemo-QoL Group, 2003), which may lead to reports of increased social

participation.

5.7 Study Limitations

Despite random sampling efforts to increase the representativeness of the sample, this study was

a voluntary study and therefore may have been susceptible to sampling bias because participants

who agreed to participate in the study may have varied in a systematic way from those who

refused to participate. However, there was a very low rate of refusal and the random sampling of

participants increased the generalizability of this study.

There may have been some element of social desirability in the participants’ responses, as the

individuals may not have wanted to report severe limitations to their healthcare team or in front

of their family members. Participants may have wanted to appear self-assured and equivalent to

their peers and thus may have reported higher scores than they truly felt. To try and account for

this, all participants were assured that their responses would be strictly confidential and

participants were encouraged to complete self-report questionnaires on their own.

Due to the cross-sectional nature of this design, the data from this study were not sufficient to

make causal inferences. However, the benefits of a cross-sectional design far outweighed the

limitations. Strengths included the need for fewer resources, no loss to follow-up or migration

bias, and minimized participant burden, as there was no follow-up period in which participants

had to be monitored.

As temporality cannot be determined when using a cross-sectional design, reverse causation bias

was possible and thus the linear models may not be valid because it may have been social

participation that exerted an effect on self-perception and not the other way around. It is

important to bear in mind that in this exploratory study, we were seeking to understand if there

was a possible relationship between self-perception, perceived social support, and social

participation, rather than trying to determine the causes of impaired social participation. We

cannot therefore deduce underlying mechanisms as it may be that social participation difficulties

lead to lower self-perception and perceived social support, or vice versa. That being said, when

exploring possible models to determine what variables may impact social participation, we found

113

that both self-perception and social support fit the model nicely and thus have been included in

this analysis.

This study was limited to English-speaking patients and this may impact the generalizability of

the results. Given that Toronto is a multi-cultural city, it would have been ideal if we could have

included participants who were not English-speaking. It is unknown if patients who do not speak

English in Toronto have different levels of social participation than their English-speaking peers,

as the questionnaires in this study were unable to assess these patients. However, this study

strived to include a representative sample of the entire hemophilia population and only four

patients were excluded from the research study due to language barriers.

The sample had a relatively high-income level and this may have skewed the results in a positive

direction, as households with higher income may have been able to afford more opportunities to

participate socially (e.g. enroll children in activities outside of school such as organized sports or

clubs). Again, given the random sampling efforts and the low rate of refusal, the generalizability

of this study to other similar settings is still good.

The small sample size increased the impact of influential observations, however the results were

essentially unchanged when influential observations were removed and the data were re-

analyzed.

Since self-perception was measured differently for children and adolescents, the sample was split

into two groups for some analyses. Although there were no significant differences found

between the two groups, the sample sizes of the split groups may have been too small to detect

any differences.

Single imputation was used to handle missing data in this study, as this was the technique

advised by all the authors of the outcome measures. More sophisticated techniques for handling

missing data such as multiple imputation may have accounted for the uncertainty of the

imputations (Stuart, 2010). However, since data was missing from just eight variables, with a

maximum of 2 participants (4.0%) missing values for any one variable, single imputation was

deemed appropriate for the analysis. Missing values appeared to be questions skipped

accidentally by participants when filling out questionnaires, and not systematically unanswered.

114

Another limitation this study faced was that the Participation Scale for Kids (i.e. the

questionnaire modification used in this study) was untested. However tests for internal

consistency of the modified measure met the minimum cut-offs recommended by Nunnally

(1978, as cited in Streiner & Norman, 2008a). Current participation scales are often targeted at

adults and those scales that did exist for children did not adequately measure social participation

in our opinion. The original Participation Scale upon which modifications were made had the

advantage of including an option for participants to select if the item was neither relevant or of

interest to him. The Participation Scale addresses the personal and subjective nature of quality of

life (Feldman, 2013) and this feature was the primary reason why this scale was selected for

inclusion in this study.

By changing some of the wording of the items on the original Participation Scale, the validity

and reliability of the scale may have been reduced. It is possible that younger children did not

fully understand the scale or the concept of peer comparison. However, modifications were

made in consultation with the author of the original scale and it was felt that the amendments

were relatively minor and that the Participation Scale for Kids still maintained face validity.

Future studies may wish to re-validate this modified scale to test the new items and determine the

scale’s psychometric properties.

Finally, the questionnaires used to measure social participation may not have adequately

assessed how much of an impact hemophilia can have on an individual. Although hemophilia

may be more of an inconvenience than a disability on a daily basis, there may be times when

hemophilia is severely limiting. For example, if a patient suffers a joint bleed he may experience

restricted mobility and the inability to engage like his peers. How can one fully embrace a

disease that is unpredictable, uncomfortable, or limiting? These scenarios do not appear to be

the norm for our participants, but they can occur and may not be fully captured by our

questionnaires. Patients with hemophilia today can live a relatively normal life, but

intermittently the disease can cause pain associated with bleeds and the burden of illness

becomes apparent.

5.8 Future Directions

With the knowledge gained from this study, future works on social participation can be

contemplated. Social participation research in the hemophilia population is very limited and is

115

often focused on adults or specifically on sport participation. It was recently noted that for

patients and family members, participation is increasingly becoming the most salient outcome

(Blanchette, O'Mahony, McJames, & Mahlangu, 2014). This study was the first step in

understanding social participation in children with hemophilia outside the realm of sport.

The next step will be to develop a children-specific scale to more accurately measure the

construct of social participation. Psychometric testing and standardization of the Participation

Scale for Kids would allow researchers to develop a better tool for assessing social participation.

Other studies may choose to explore the construct of social participation at a cross-cultural level.

The Participation Scale for Kids could be administered to samples of patients in different

countries to see what social participation is like in other parts of the world. Our sample had few

patients with arthropathy or co-infection, but this is not necessarily the case for hemophilia

patients around the world. Indeed, the Participation Scale for Kids is currently being

administered at some centers in São Paulo, Brazil, where prophylaxis has only recently been

used more readily.

Future works may also include administering the Participation Scale for Kids to children with

and without chronic illnesses to compare patients with hemophilia to others. Such a study would

inform efforts to understand what other contextual factors are related to social participation and

if children with other chronic illnesses have levels of social participation on par with their peers.

Longitudinal studies that assess temporality may be able to isolate factors that directly contribute

to social participation. With the knowledge gained from such studies, interventional studies may

be developed to attempt to effect change on social participation.

5.9 Conclusions

In conclusion, the data show that boys with hemophilia have minimal social participation

restrictions, adequate self-perception, good social support, and their illness has minimal impact

on the family. This study is the first, to our knowledge, that has examined social participation

and its relationship to self-perception, social support, and the impact of the disease on the family

in boys with hemophilia.

116

In keeping with the ICF model, the data illustrate that the interaction between personal and

environmental factors as well as the health condition of hemophilia are associated with one’s

social participation. The data show that self-perception and social support were moderately to

strongly correlated with social participation. This study shows that participants who have higher

levels of self-perception and social support will also report higher levels of social participation.

Although we do not know the directionality of this relationship, it would seem appropriate to

encourage patients to take part in activities to increase self-perception, strengthen their social

support network, and increase their social participation. Doing so may result in an increase in

one or more of these domains, all of which would increase the patient’s health-related quality of

life.

Psychologists have been identified as crucial members in the comprehensive care team, as they

can assist patients with the self-management of their disease (Coppola et al., 2011) and

consequently foster the development of the patient’s independence. Monitoring for depression in

patients is important because health outcomes can be compromised when patients are depressed,

leading to increased risk taking behaviors or poor treatment compliance (Iannone et al., 2012).

Incorporating routine screening into usual care for hemophilia may allow comprehensive care

teams to easily identify patients at higher risks for low self-worth. Indeed, St. Michael’s

Hospital in Toronto, Canada, has rolled out such an intake assessment in its hemophilia clinic

and depression screening has been tested in Tucson, United States (Iannone et al., 2012).

Identifying patients who struggle with depression or low self-worth will enable healthcare

providers to offer appropriate support to the patients.

117

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Appendices

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Appendix A: Research Ethics Board Approval Letters

Copy of Approval by The Hospital for Sick Children

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127

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Copy of Approval by the University of Toronto

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Appendix B: Data Collection Instruments

Copy of Participation Scale for Kids (modified)

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Copy of Self-Perception Profile for Children

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Copy of Self-Perception Profile for Adolescents

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Copy of Social Support Scale for Children

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Copy of PedsQL™ Family Impact Module

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140

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Appendix C: Family Situation Family Situation (N = 50) Variable n (%) Marital Status

Married/Common-law Separated Unmarried Divorced

41 (82.0)

4 (8.0) 2 (4.0) 3 (6.0)

Primary Guardian Mother & Father Mother Father

37 (74.0) 12 (24.0)

1 (2.0) Living Area

Rural Urban

Household Income Low (less than CAD$30,000) Medium (CAD$30,000-$80,000) High (greater than CAD$80,000)

7 (14.0)

43 (86.0)

6 (12.2) 17 (34.7) 26 (53.1)

Appendix D: Treatment

Table D1 Treatment Modalities Variable n (%) Where treatment occurs

Home Clinic or Hemophilia Treatment Center Other

37 (74.0) 10 (20.0)

3 (6.0) Who infuses treatment

Mother Father Mother & Father Self-Infusion Clinic staff Mother & Self-Infusion Mother, Father & Self-Infusion

15 (31.3)

4 (8.3) 4 (8.3)

8 (16.7) 12 (25.0)

4 (8.3) 1 (2.1)

Treatment frequency within the last 6 months On-demand Once per week 2 times per week 3 times per week Every other day Every day Immune tolerance induction or bypassing agents

12 (24.0)

3 (6.0) 13 (26.0)

3 (6.0) 18 (36.0)

1 (2.0) 0 (0.0)

Table D2 describes the treatment differences between moderate and severe participants.

Participants with moderate severity were more likely to receive on-demand therapy than severe

participants. If moderate participants required prophylaxis, it was far more likely to be

secondary in nature. On the other hand, participants with severe phenotype were less likely to

receive on-demand therapy as the majority of participants were on primary or secondary

prophylaxis.

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Table D2 Frequency of treatment over entire life by participant factor level (N = 50) Variable Moderate (n (%)) Severe (n (%)) On-demand

No Yes

2 (13.3)

13 (86.7)

22 (62.9) 13 (37.1)

Primary prophylaxis No Yes

14 (93.3)

1 (6.7)

19 (54.3) 16 (45.7)

Secondary prophylaxis No Yes

11 (73.3) 4 (26.7)

19 (54.3) 16 (45.7)

Short-term prophylaxis No Yes

15 (100.0)

0 (0.0)

33 (94.3)

2 (5.7)

Appendix E: Internal Consistency Measures of Reliability for Social Participation Scale for Kids Raw Variables Standardized Variables Item Item-Total Correlation Cronbach’s alpha Item-Total Correlation Cronbach’s alpha 1 0.1 0.7 0.1 0.7 2 0.3 0.7 0.2 0.7 3 -0.0 0.7 0.0 0.7 4 0.1 0.7 0.1 0.7 5 -0.0 0.7 0.0 0.7 6 0.6 0.7 0.5 0.7 7 0.5 0.7 0.5 0.7 8 0.7 0.7 0.6 0.7 9 0.3 0.7 0.2 0.7 10 0.5 0.7 0.4 0.7 11 0.3 0.7 0.4 0.7 12 0.4 0.7 0.4 0.7 13 0.2 0.7 0.3 0.7 14 0.1 0.7 0.1 0.7 15 0.2 0.7 0.2 0.7 16 0.5 0.7 0.4 0.7 17 0.4 0.7 0.4 0.7 18 0.3 0.7 0.3 0.7 19 0.3 0.7 0.3 0.7 20 0.3 0.7 0.3 0.7

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Appendix F: Item Responses for Social Participation Scale for Kids

See Appendix B for Social Participation Scale for Kids items.

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 1

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 2

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 3

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 4

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 5

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 6

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 7

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 8

144

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 9

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 10

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 11

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 12

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 13

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 14

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 15

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 16

145

Appendix G: Reference Scores from the Manual for the Self-Perception Profile for Children

(Harter, 1985a) Reference Scores for Self-Perception for Sixth Grade Boys Sample Social Acceptance (M (SD)) Athletic Competence (M (SD)) Global Self-Worth (M (SD)) A (n = 206) 3.1 (0.6) 3.2 (0.6) 3.2 (0.6)

Appendix H: Social Support Scale for Children Scores by Age Group Pearson Correlations between Social Participation and Social Support by Age Group Variable Children Adolescents

Parental Support/Regard -0.8*** -0.2 Teacher Support/Regard -0.7*** -0.4 Classmate Support/Regard -0.7*** -0.3 Close Friend Support/Regard -0.5** -0.4 Note. *Correlation is significant at p < 0.05. **Correlation is significant at p < 0.01. ***Correlation is significant at p < 0.001.

Appendix I: Reference Scores from the Manual for the Social Support Scale for Children

(Harter, 1985b) Reference Scores for Social Support for Sixth Grade Boys Sample Parent (M (SD)) Teacher (M (SD)) Classmate (M (SD)) Close Friend (M (SD)) Elementary (n = 35) 3.0 (0.8) 2.9 (0.7) 2.9 (0.7) 2.9 (0.6)

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 17

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 18

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 19

0 10 20 30 40 50

0 1 2 3 5 Freq

uenc

y

Score

Item 20

146

Appendix J: Comparison with Reference Scores Comparison with Reference Scores for the Social Support Scale for Children Subscale t df p Parental Support/Regard 4.0 53.1 0.0002 Teacher Support/Regard 3.0 77.1 0.004 Classmate Support/Regard 4.3 67.2 <0.0001 Close Friend Support/Regard 4.9 74.9 <0.0001

Appendix K: Normality of Residuals Normality of Residuals Variable Skewness Kurtosis Shapiro-Wilk Self-Perception Profile for Children/Adolescents Social Acceptance

Children & Adolescents (n = 50) Children (n = 30) Adolescents (n = 20)

1.0 0.6 0.9

0.9 0.2

-0.6

0.9*** 0.9** 0.8**

Athletic Competence Children & Adolescents (n = 50) Children (n = 30) Adolescents (n = 20)

1.9 1.4 0.8

3.9 1.5

-0.2

0.8*** 0.8***

0.9* Global Self-Worth

Children & Adolescents (n = 50) Children (n = 30) Adolescents (n = 20)

1.1 1.4 0.7

1.6 3.3

-0.8

0.9** 0.9** 0.9*

Social Support Scale for Children Parental Support/Regard (n = 50) 0.3 1.0 1.0 Teacher Support/Regard (n = 50) 0.7 0.4 0.9** Classmate Support/Regard (n = 50) 0.5 1.0 0.9* Close Friend Support/Regard (n = 50) 1.3 1.7 0.9*** PedsQL™ Family Impact Module (n = 50) Total Score 1.9 4.1 0.8*** Physical Functioning 1.8 3.8 0.8*** Emotional Functioning 1.8 4.0 0.8*** Social Functioning 1.9 4.1 0.8*** Cognitive Functioning 1.7 3.6 0.8*** Communication 1.7 3.4 0.8*** Worry 1.6 2.6 0.8*** Daily Activities 1.9 4.2 0.8*** Family Relationships 1.7 3.0 0.8*** Note. *Normality test is significant at p < 0.05. ** Normality test is significant at p < 0.01. *** Normality test is significant at p < 0.001.

147

Appendix L: Residual Plots

Residual plots for Social Participation and Self-Perception

Figure L1. Social Acceptance

Figure L2. Athletic Competence

148

Figure L3. Global Self-Worth

Residual plots for Social Participation and Social Support

Figure L4. Parental Support/Regard

149

Figure L5. Teacher Support/Regard

Figure L6. Classmate Support/Regard

150

Figure L7. Close Friend Support/Regard

Residual plots for Social Participation and Family Impact

Figure L8. Total Score

151

Figure L9. Physical Functioning

Figure L10. Emotional Functioning

152

Figure L11. Social Functioning

Figure L12. Cognitive Functioning

153

Figure L13. Communication

Figure L14. Worry

154

Figure L15. Daily Activities

Figure L16. Family Relationships

155

Appendix M: Non-Parametric Tests

Self-Perception

When the data was re-analyzed using Spearman’s rank-order correlation coefficients, a strong

correlation was found between social participation and Social Acceptance (rho = -0.6, p = <

0.001), when looking at the scores of both children and adolescents. Moderate correlations were

found between social participation and Athletic Competence (rho = -0.4, p = < 0.01) and Global

Self-Worth (rho = -0.4, p = < 0.01).

When examining Children alone, social participation was strongly associated with Social

Acceptance (rho = -0.6, p = < 0.001) and Global Self-Worth (rho = -0.6, p = < 0.001), and not

significantly correlated with Athletic Competence (rho = -0.3, p = > 0.05). For adolescents, a

strong correlation was found between social participation and Athletic Competence (rho = -0.5, p

= < 0.05), but no statistically significant relationship was found between social participation and

Social Acceptance (rho = -0.4, p = > 0.05) or Global Self-Worth (rho = -0.2, p = > 0.05). Both

the Spearman and Pearson correlations are listed in Table M1.

These relationships suggested that overall children and adolescents with more adequate levels of

self-perception tended to have fewer social participation restrictions.

Table M1 Correlations between Social Participation and Self-Perception Variable Spearman rho Pearson r Social Acceptance

Children & Adolescents Children Adolescents

-0.6*** -0.6***

-0.4

-0.5*** -0.5**

-0.3 Athletic Competence


-0.4**

-0.3 -0.5*

-0.2 -0.1

-0.6** Global Self-Worth


-0.4**

-0.6*** -0.2

-0.6*** -0.7***

-0.3 Note. *Correlation is significant at p < 0.05. **Correlation is significant at p < 0.01. ***Correlation is significant at p < 0.001.

Social Support

Spearman rank-order correlations continued to find correlations between social participation and

social support (see Table M2). There was a strong correlation between social participation and

support/regard from classmates (rho = -0.5, p = < 0.001), but only moderate correlations between

156

social participation and support/regard from parents (rho = -0.4, p = < 0.01), close friends (rho =

-0.4, p= < 0.01), and teachers (rho = -0.3, p = < 0.05).

These correlations show that children and adolescents who reported more social support from

their parents, classmates, teachers, and close friends tended to report fewer social participation

restrictions.

Table M2 Correlations between Social Participation and Social Support Variable Spearman rho Pearson r Parent -0.4** -0.6*** Teacher -0.3* -0.5*** Classmate -0.5*** -0.6*** Close Friend -0.4** -0.4** Note. *Correlation is significant at p < 0.05. **Correlation is significant at p < 0.01. ***Correlation is significant at p < 0.001.

Family Impact

Much like the Pearson correlations, few associations were observed between social participation

and family functioning. There continued to be a moderate correlation between social

participation and social functioning (rho = -0.3, p = < 0.05), and there was also a moderate

correlation between social participation and daily activities (rho = -0.3, p < 0.05). These

correlations indicated that parents/guardians who reported less impairment in social functioning

and daily activities would in turn have children who were less likely to report social participation

restrictions (see Table M3).

Table M3 Correlations between Social Participation and Family Impact Variable Spearman rho Pearson r Total Score -0.2 -0.2 Physical Functioning -0.2 -0.2 Emotional Functioning -0.3 -0.2 Social Functioning -0.3* -0.3* Cognitive Functioning -0.2 -0.2 Communication -0.1 -0.2 Worry -0.1 0.0 Daily Activities -0.3* -0.3 Family Relationships -0.2 -0.2 Note. *Correlation is significant at p < 0.05.

157

Appendix N: Sensitivity Analysis Minimum and Maximum Imputed Values for Missing Data Variable Minimum r Maximum r Self-Perception Profile for Children/Adolescents Social Acceptance


-0.5*** -0.6***

-0.3

-0.5*** -0.5**

-0.3 Athletic Competence


-0.2 -0.2

-0.6**

-0.2 -0.1

-0.6** Global Self-Worth


-0.6*** -0.7***

-0.3

-0.6*** -0.7***

-0.3 Social Support Scale for Children Parental Support/Regard -0.6*** -0.6*** Teacher Support/Regard -0.5*** -0.5*** Classmate Support/Regard -0.6*** -0.6*** Close Friend Support/Regard -0.4** -0.4** PedsQL™ Family Impact Module Total Score -0.2 -0.2 Physical Functioning -0.2 -0.2 Emotional Functioning -0.2 -0.2 Social Functioning -0.3 -0.3 Cognitive Functioning -0.2 -0.2 Communication -0.2 -0.2 Worry 0.0 -0.0 Daily Activities -0.3* -0.3 Family Relationships -0.2 -0.2 Note. *Correlation is significant at p < 0.05. **Correlation is significant at p < 0.01. ***Correlation is significant at p < 0.001.

158

Appendix O: Model Checking for Self-Perception

Figure O1. Histogram for Residuals

Figure O2. Q-Q Plot for Residuals

159

Appendix P: Model Checking for Social Support

Figure P1. Histogram for Residuals

Figure P2. Q-Q Plot for Residuals

160

Appendix Q: Linear Modeling

Table Q1. Parameter Estimates for Linear Regression Parameter Estimates with Age and Factor Level as Predictor Variables Social Participation Predictor Parameter Estimate Standard Error Test Statistic p-value Omnibus F-test (F(ndf,ddf)) 2.8 (2, 47) 0.1 Age -0.7 0.3 -2.4 0.0 Factor Level -0.3 1.8 -0.2 0.9 Self-Perception: Social Acceptance Predictor Parameter Estimate Standard Error Test Statistic p-value Omnibus F-test (F(ndf,ddf)) 2.4 (2, 47) 0.1 Age 0.1 0.0 2.2 0.0 Factor Level 0.1 0.2 0.4 0.7 Self-Perception: Athletic Competence Predictor Parameter Estimate Standard Error Test Statistic p-value Omnibus F-test (F(ndf,ddf)) 0.0 (2, 47) 1.0 Age 0.0 0.0 0.3 0.8 Factor Level 0.0 0.2 0.0 1.0 Self-Perception: Global Self-Worth Predictor Parameter Estimate Standard Error Test Statistic p-value Omnibus F-test (F(ndf,ddf)) 0.8 (2, 47) 0.5 Age 0.0 0.0 0.9 0.4 Factor Level 0.2 0.2 0.9 0.4

None of the above models met overall significance with an omnibus F-test and therefore

individual covariates were not further explored.

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Social Participation, Self-Perception, and Social …...ii Social Participation, Self-Perception, and Social Support in Boys Living with Hemophilia Aubrey Serah Chiu Master of Science