Unraveling patient‐preferred health and treatment outcomes ... · DOI 10.1002/acr.33834 This article has been accepted for publication and undergone full peer review but has not

Running head: Patient-preferred outcomes in early rheumatoid arthritis

Title: Unraveling patient-preferred health and treatment outcomes in early

rheumatoid arthritis: a longitudinal qualitative study

Kristien Van der Elst, RN, MSc,1-3 Sabrina Meyfroidt, MSc,2 Diederik De Cock, MSc,2 An De

Groef, MSc,4 Els Binnard,4 Philip Moons, RN, PhD,3,5,6 Patrick Verschueren, MD, PhD,1,2 and

René Westhovens, MD, PhD1,2

1University Hospitals Leuven, Department of Rheumatology, B-3000 Leuven, Belgium

2KU Leuven - University of Leuven, Department of Development and Regeneration, Skeletal

Biology and Engineering Research Center, B-3000 Leuven, Belgium

3KU Leuven - University of Leuven, Department of Public Health and Primary Care,

Academic Center for Nursing and Midwifery, B-3000 Leuven, Belgium

4Patient Research Partners, B-3000 Leuven, Belgium

5University of Gothenburg, Institute of Health and Care Sciences, SE-405 30 Gothenburg,

Sweden

6Copenhagen University Hospital, The Heart Center, DK-2100 Copenhagen, Denmark

Corresponding author: Kristien Van der Elst, Department of Rheumatology, University

Hospitals Leuven, Herestraat 49, B-3000 Leuven, Belgium; Tel: +32-16-342541; Fax: +32-

16-342543; E-mail address: [email protected]

Funding: This work was supported by Fonds Wetenschappelijk Reuma Onderzoek (FWRO).

The CareRA study (EudraCTnumber: 2008-007225-39) is funded by a Flemish governmental

grant (Agency for Innovation by Science and Technology [IWT]). Patrick Verschueren holds

the Pfizer chair for early rheumatoid arthritis management at the KU Leuven.

Word count: 3800

Original Article Arthritis Care & ResearchDOI 10.1002/acr.22824

This article has been accepted for publication and undergone full peer review but has not beenthrough the copyediting, typesetting, pagination and proofreading process which may lead todifferences between this version and the Version of Record. Please cite this article as an‘Accepted Article’, doi: 10.1002/acr.22824© 2015 American College of RheumatologyReceived: Mar 25, 2015; Revised: Sep 09, 2015; Accepted: Dec 15, 2015

This article is protected by copyright. All rights reserved.

2

Abstract

Objectives: To unravel the perspective of patients with early rheumatoid arthritis (ERA) on

preferred health and treatment outcomes at two time points during the early stage of their

disease and treatment.

Methods: In a longitudinal, qualitative, explorative study, we individually interviewed 26

patients with ERA 4-6 months after start of the initial rheumatoid arthritis (RA) treatment.

Fourteen of these participants took part in 1 of 3 focus groups at least one year after

treatment initiation. Interviews were audiotaped, transcribed verbatim and analyzed using the

constant comparison method. Two patient researchers completed the interdisciplinary

research team.

Findings: Patients with ERA ultimately strive to be normal again, literally and figuratively.

Outcome preferences inherent to this urge for normality were related to aspects of disease

control, physical aspects, aspects of participation and mental aspects. Initially, patient

outcome preferences in ERA were primarily related to pain relief, medication side effects and

burden, and emotional well-being. Patient-preferred outcomes evolved over the ERA disease

course, with subtle changes in terminology used by participants and with pain relief staying in

the foreground.

Conclusion: From a patient perspective, normality is the ultimate outcome to target in ERA.

Our study produced knowledge for designing more targeted therapeutic interventions aimed

at normalizing patients’ health and life in all its aspects during a crucial phase of RA.

Page 2 of 35

John Wiley & Sons, Inc.

Arthritis Care & Research


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Significance and Innovations

• Striving for shared decision making between health-care professionals and patients at the

earliest stage of care is imperative to achieve effective and efficient rheumatoid arthritis

management.

• Longitudinal qualitative research in patients with early rheumatoid arthritis reveals the

dynamics in patients’ outcome preferences pointing to “normality” as a key target.

• These findings should be applied in order to optimize care in a crucial disease phase, by

enhancing early interventions, which in turn may prevent long-term disease

consequences.

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Introduction

Despite effective pharmacological treatments available, patients with rheumatoid arthritis

(RA) continue to report unmet needs, and the impact on the individual and society remains

considerable (1, 2). This care gap suggests that patients’ health and treatment preferences

are not sufficiently understood and met. Outcome assessment in RA has already changed

profoundly in that the patient’s perspective and patient-reported outcomes (PROs) are now

being considered. However, the question remains which outcomes to evaluate and how to

measure them, with maybe different answers from the patients’ and health-care

professionals’ perspective (3, 4).

The importance of responding to patients’ needs and preferences cannot be overstated in

chronic conditions (4, 5). Patient preferences are molded by their perspectives, beliefs,

expectations, and goals for health and life (6). Health-care professionals and patients share

common treatment goals. However, additionally targeting patient-preferred outcomes may

improve patients’ treatment adherence and satisfaction, leading to better health outcomes

(7). This patient-centered focus has been acknowledged as a priority health-care quality

dimension (8).

Not all PROs are patient-centered and conversely, often not all outcomes that truly matter to

patients are measured (9). Qualitative studies on the patient perspective in RA have

challenged outcomes and outcome core sets held by health-care professionals, discovering

that patient preferences could go beyond the traditional RA measures of disease activity and

severity (10-12). None of these cross-sectional qualitative studies focused on what matters

specifically to patients with early RA (ERA), while outcome preferences can differ depending

on disease duration (13). Such early changes in health perceptions and preferences find a

conceptual basis in for example the chronic illness trajectory framework that describes,

depending on the stage in an individual’s illness trajectory, dynamics in illness experiences,

self-perceptions, expectations, and care needs (14).

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Although there is no clear definition of ERA from the patient perspective in literature, the

early disease stage is probably the most precarious period for patients, because in this stage

they lack disease-related experience, knowledge, and awareness (15). Moreover, treating

RA during its earliest stage likely increases chances for positive, long-term outcomes (16).

Since perceptions and expectations of newly diagnosed patients may change during the

overwhelming and rapidly evolving early disease stage, a longitudinal perspective would

expand our understanding on outcome preferences in ERA. This study aims to prospectively

unravel the perspective of patients with ERA on preferred health and treatment outcomes.

Patients and Methods

Study design

We performed a longitudinal, qualitative, explorative study in collaboration with two patient

research partners with RA (AG, EB) (17). Participants were invited to be interviewed twice

over a one-year period in the ERA phase: once in an individual interview (t1) and once in

focus groups (t2).

We reasoned that conducting individual interviews at t1 was preferable, because sharing

personal opinions in a group setting could be delicate for recently diagnosed patients, as

confirmed by our patient researchers. These interviews were conducted 4-6 months after

treatment initiation, which should be sufficient time for diagnostic procedures to be finalized

and RA therapy to be ongoing. Furthermore, we assumed that after 4-6 months participants

would be better able to grasp different disease and treatment aspects and communicate

about their outcome preferences. At t2, focus groups were considered to be preferable, as

group interactions encourage persons to share their thoughts and beliefs, which can result in

more and varied data (18). Furthermore, focus groups provide views other than those

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provided by individual interviews, which also possibly enriches the data (19, 20). Conducting

the focus groups at least one year after treatment initiation helped participants to develop

disease perspective and to feel more comfortable in sharing their story. Participants would by

then probably have already informally interacted with peers.

Context

The present study was embedded in the Care for early RA (CareRA) trial; a prospective two-

year, investigator-initiated, multicenter, randomized controlled trial rooted in daily practice,

which compares initial treatment combinations in ERA (21). Patients fulfilled the American

College of Rheumatology 1987 classification criteria for RA (22), and had a disease duration

of ≤ 1 year.

Recruitment of interview participants and composition of focus groups

Patients included in the CareRA trial before January 2012 could be invited by their

rheumatologist for an individual interview if this could be scheduled within 4-6 months after

treatment initiation. From the list of 46 eligible patients, participants were purposively

sampled based on type of rheumatology practice and geographic region. Patients obliged or

deciding to discontinue RA treatment were also considered.

Twenty-six patients were interviewed at t1, and all of these were invited to participate in the

subsequent focus groups, except one who withdrew trial consent. Seven interviewed

participants declined to engage in a focus group because they either did not want to share

their experiences, felt uncomfortable in a group or were afraid to face their peers. Four

participants later declined their group participation because of scheduling conflicts.

Eventually, three focus groups were held, with a total of 14 participants. A flow chart of

participant recruitment is displayed in figure 1. Table 1 presents the well-balanced participant

characteristics at both measurement points.

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The Human Research Ethics Committee of the University Hospitals Leuven approved this

study; participants provided written informed consent (23).

Data collection

Between April and August 2012, KE and SM conducted face-to-face, semi-structured

individual interviews at the participants’ home or at the rheumatology clinic (their preference).

The interview structure is presented in table 2. Interviews lasted about 45 minutes.

In March 2013, KE conducted focus groups at non-clinical locations, with SM observing

group interactions. After discussions with the patient researchers, we opted to use a modified

nominal group technique to structure the focus groups and stimulate interactions (24). Focus

groups consisted of three rounds (Table 2) and lasted about one hour.

The interview guides were developed with the help of our patient researchers and contained

open-ended and probing questions (Table 2). Based on the insights gained from the first

interviews, we added three questions to the t1 interview guide to enrich data collection (25-

27). Interviews were audiotaped and transcribed verbatim. Observational field notes were

documented for interviews and focus groups; these were related to setting, participants’

behavior, interaction between participants, and remarkable quotations. These notes were

used as contextual information when analyzing and interpreting the data (20). Participants

completed a pre-interview questionnaire documenting sociodemographic and clinical

information. After the interviews, they self-reported their general health, level of pain, and

fatigue during the past week on a visual analog scale (VAS) (Table 1).

Data analysis

Data analysis and collection were performed concurrently, with analysis beginning

immediately after the first interview. Qualitative Analysis Guide of Leuven (QUAGOL) with

the constant comparison method was used to analyze individual interviews and focus groups

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(26). The two patient researchers were involved in the entire analysis process, helping the

researchers to understand and explain the data.

Interview transcripts were independently analyzed by two researchers (KE, SM) and the two

patient researchers. They read each transcript several times to grasp its essentials. For each

interview, fragments deemed relevant for answering the research question by each analyst

were extracted and coded; similar codes were grouped and sorted into potential themes.

Insights from new interviews were constantly used to further uncover and refine emerging

themes and subthemes. Consensus on the individual coding was reached by the analysts,

and this master list of codes was entered in NVivo 10® by the researchers.

After the data were reviewed and coded, we adopted Saldaña’s guiding questions for

analyzing longitudinal data (Table 3) (28). The meaning and phrasing of codes and themes

were discussed in peer debriefings, which included input from patient researchers,

rheumatologists, nurses, and clinical researchers.

Findings

At t1 and t2, participants’ views on preferred outcomes were related to four aspects inherent

to the overarching outcome of a return to being normal: (1) aspects of disease control, (2)

physical aspects, (3) aspects of participation, and (4) mental aspects (Figure 2). Table 4

shows example quotes illustrating the findings.

Return to being normal

At t1, participants were generally set on returning to their normal life as soon as possible and

this in its narrowest sense. They described a struggle to stay normal and hoped to re-

establish their former health, not constantly being aware that they have RA. At t2, participants

were more subtle and nuanced in their normality expressions, and mentioned now

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preferences like “remaining bodily well” and “controlling my RA”. Yet, they still preferred to

have a life as normal as possible. Participants also referred to their ‘healthy’ peers, wanting

to grow old in a comparable way. Normality was a personal construct, shared among all

participants.

Aspects of disease control

Proof of disease control

Particularly at t1, participants expressed the need for visual feedback about test results in

order to objectify their disease evolution. When being ‘obliged’ to take more medication, they

hoped that this would at least have a clear positive effect on their health. This proof of

disease control provided reassurance. At t2, they still wanted such proof but less strongly.

Prevention or stabilization of joint damage

Participants mentioned joint damage more frequently at t1—especially those knowing

someone with joint malformations—because they merely perceived it as a worst-case

scenario of having ‘rheumatism’. At t2, participants’ initial fear of and focus on joint damage

diminished.

Less medication

Less medication was a dominant outcome preference at t1, a time when most participants

had just been prescribed a combination of disease-modifying antirheumatic drugs with a

glucocorticoid-bridging scheme. A reduction in medication—inherent to the study protocol—

was viewed as health improvement proof. Participants feared for long-term adverse effects of

medication and believed a reduction would limit these. They also struggled mentally with a

sudden need for drugs after previously being ‘healthy’. They hoped to discontinue RA

therapy, although this was considered unlikely.

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At t2, taking medication was still viewed as unpleasant, but participants expressed less

resistance and perceived it more as necessary for disease control, which they considered

more important than having to take medication. However, less medication was still

considered preferable because of side effect concerns.

Physical aspects

Relief of pain and other physical symptoms

At t1 and t2, participants identified pain relief as a major outcome preference, surpassing their

preference to be relieved of other physical symptoms, such as fatigue, swelling, and

stiffness. Some preferred to be symptom-free, while others were satisfied with a lower level

of symptoms. Participants were eager to start treatment, especially for pain. They preferred

immediate pain relief and this was experienced as a reassuring and promising treatment

start.

At t2, pain relief, especially the ‘intolerable’ pain related to flares, remained a highly preferred

outcome, despite perceived disease control. Participants feared their initial pain experience

and remembered its impact on daily life. They identified pain as an outstanding symptom and

focused less on other symptoms. Physical symptoms were regarded more as the inevitable

consequences of RA, reflected in expressions like “a flare can occasionally be expected”.

Relief of fatigue was not singled out anymore at t2, despite participants reporting comparable

VAS fatigue levels at t1 and t2 (Table 1).

Improved joint function and mobility

Especially at t1, participants preferred to be able to do basic functions themselves again (e.g.,

opening cans, taking stairs), which were self-evident before. This preference underscored

their diminished strength, grip, fine motor skills, and mobility, abilities that needed to be

restored.

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At t2, participants were more focused on social participation, in general, except in cases of

perceived “setbacks”; then, they focused on regaining a basic level of physical functioning.

Limited side effects

At t1, participants’ preference to limit side effects of RA medication were a main concern,

because these affected their sense of normality (e.g., weight gain created frustrations and

shame; nausea limited social participation), or because of being biased against the

prescribed medication. Furthermore, they feared but hoped not to compromise their long-

term health by taking such “strong” drugs.

At t2, their initial fear decreased, and although side effects were still of concern, these were

more accepted. New expressions, such as “no additional side effects” and “feeling more

comfortable with side effects” emerged. Participants weighted to a higher extent the benefits

of treatment against its side effects. Concerns about the long-term impact of medication,

however, remained present.

Improved sleep

At t1 and t2, improved sleep was of concern to participants who experienced or feared a

significant impact of pain on their sleep, which was specifically mentioned as a reason why

pain relief remained a highly preferred outcome at t2.

Aspects of participation

Performing activities of daily living

At t1, participants mainly focused on self-care and domestic activities. Their minds were fixed

on doing everyday activities normally: autonomously, spontaneously, and at the same pace

and ease as before. Loss of autonomy was a main cause of frustration, and extremely

important for them to regain.

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At t2, participants came more to terms with a shifted performance state, such as more readily

accepting help and trying to pace their activities. Nevertheless, their goal remained to regain

normal participation.

Engaging in work and/or leisure

At t1 and t2, participants expressed the importance of being active again, remaining involved

in social activities and continuing hobbies. They feared being excluded from social activities,

or being unable to engage fully. Employed work was especially important at t1, because they

did not want to fail at their previously mastered tasks. Work became less important over time

for those experiencing ongoing disease impact.

Fulfilling family, social, and/or societal roles

Participants expressed the need to fulfill their normal roles and activities again in the family

and with friends. A key concern was whether they could keep up with relatives, friends, and

colleagues.

Family and social roles were discussed more at t2. In general, limited change in role

preferences was expressed. For the youngest participant, her future role as a mother

became more apparent because she experienced limited improvement.

Vitality

At t1, regaining vitality was a preferred outcome among those unable to participate in daily life

as before. Participants talked about needing more physical energy and regaining the mental

drive to start and pursue activities. Some mentioned vitality and fatigue together as being

closely related. At t2, participants did not specifically discuss vitality.

Mental aspects

Emotional well-being

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The RA diagnosis was shocking and led to uncertainty about what to expect from having

‘rheumatism’. At t1, participants were still emotionally overwhelmed. Hope remained the

strongest emotion, both for the present and future (e.g., hope that treatment would have

lasting effect). At t1 and t2, the experience of pain persistency, disease progression and the

need to adapt treatment (which was associated with a lack of improvement), led to doubts

and additional mental distress. Regaining their emotional well-being was underscored more

at t1 as being important for themselves and for reassuring their significant others. Other

shared preferences were having happy relationships and understanding from family and

friends. Generally, participants described a more significant psychological impact during the

early phase and their disease-related emotions and cognitions changed later in the disease

course.

Self and identity

Suddenly having to rely on others at a young age, failing at simple daily tasks, diminished

family and social roles, and feeling like a burden to others affected participants’ self-esteem.

They referred to their pre-RA identity, comparing it to their current one and longing to regain

a normalized sense of self.

Life enjoyment

At t1, participants preferred to have more life enjoyment, which was altered by their current

disease concerns. At t2, their mental outlook was more positive; they seemed to enjoy the

daily things in life again, despite RA.

Not feeling ill

Particularly at t1, participants did not want to feel ill, especially when being confronted with

physical symptoms, disabilities, and taking medication. They did not want to be patronized or

treated like a sick person. For those who felt well again, taking medication became a daily

confrontation with RA.

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Discussion

We prospectively unraveled, for the first time, the health and treatment outcome preferences

of patients with ERA. Our study showed shifting outcome preferences in the early stages

after RA diagnosis and treatment initiation, with “normality” as overarching theme.

As with previous qualitative studies on the patient perspective (10-13, 29-32), normality was

a prominent theme in our study. For example, Sanderson et al. distinguished six normality

typologies in RA (33). However, despite the obvious similarities, these findings in RA and

other chronic conditions involved patients in a different context, making them challenging to

compare with our findings derived prospectively in patients at an early disease stage.

Our patients with ERA initially conceptualized this normality as regaining the health and life

they had before RA, as soon as possible. Thus, they preferred a rapid improvement

(especially for pain). This gave them hope and helped them to deal with fear and uncertainty.

These are meaningful discoveries, as new patients gradually shape their understanding and

attitude toward RA, but initially lack experience and illness identity. Regarding adjustment to

RA, the role of illness perceptions has been described previously (34), whereas patients who

received a quick diagnosis and effective medical treatment perceived their life to be

unaffected by RA (35). This is consistent with Kristiansen et al.’s findings that patients in

early clinical remission continue to experience living a normal life (36). These findings,

together with our data support the view that, aside from its importance when targeting

classical disease outcomes in ERA, time could also be crucial from the patient perspective.

Perhaps, the present findings point to a new opportunity for prevention. Health-care

professionals should then endeavor to meet patients’ initial normality construct in all its

aspects, to help them adjust to RA in a healthy way, and to intervene before “illness-as-

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burden beliefs” get rooted (37, 38). Nurses can be seen as pivotal in providing such

necessary psychosocial care (39, 40).

We observed subtle changes over time in how normality was shaped. Participants developed

a more realistic and coherent view (e.g., symptom normalization, medication as being

necessary at the moment) having passed that particular acute disease phase, which fits with

theoretical models on chronic illness management (14, 37). This altered view made their

initial pre-RA normality shift to a normality that was not completely as going back to their pre-

disease life. This was especially the case for those who perceived an unsatisfactory

treatment response. Nevertheless, at the second time point, participants remained close to

their preferred pre-RA normality like recently after diagnosis. The notion of normality in the

early disease stage should be further developed and studied in other settings and cultures.

Over time, participants did not report new preferred outcomes. Pain relief remained a

predominant outcome preference, as in previous RA research (41, 42). Unexpectedly, fatigue

relief was rarely mentioned later in the ERA disease course, even though it has been

proposed as a patient-preferred outcome in late RA (43). The apparent discrepancy between

relatively stable reporting of fatigue by VAS over time and the decreasing importance

reflected in the focus groups needs more study. Other outcomes that were less or no more

mentioned over time were related to proof of disease control, joint damage prevention, sleep

quality, vitality, self and identity, life enjoyment, and not feeling ill. Moreover, changes in

participants’ perspective started to emerge with a focus beyond treatment issues. These

dynamics points to the continued need for longitudinal studies to understand patient

expectations in different stages of their disease, to successfully adapt RA care processes

(14).

Our findings provide a framework for promoting effective communication between health-

care professionals and new patients based on shared goals and understanding (15, 44, 45).

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Patient-centered information exchange is key in constructing such partnership (46). In ERA,

health-care professionals should include normality in their conversations; explore what this

normality means to each patient and discuss the management of potential threats to it.

Moreover, this study learned us that newly diagnosed patients with RA prefer a rapid and

prolonged treatment response, preferably without side effects and without chronic medication

use. These findings emphasize the importance of addressing an immediate pain-reducing

effect, coping with pain and side effects, potential side effects, and prospects of drug-free

remission and medication tapering. Patient empowerment and self-advocacy could then be

established early in the chronic disease course (47).

Health-care professionals also need to remember that patient-preferred outcomes in ERA

can be dynamic and need repeated evaluation. For example, aspects of participation should

be discussed more into depth later on, and the altered focus on joint damage prevention

underscores the need for ongoing patient education on RA treatment targets.

Employing individual interviews and focus groups at different times produced methodological

challenges. Firstly, not all participants of the individual interviews (t1) were willing to take part

in the focus groups (t2). Using repeated interviews might have increased participation. We

invited participants for the future focus group immediately after the interview; perhaps, they

would have reacted more positively if invited later. Nevertheless, the fact that some

participants felt uncomfortable to participate in a group setting supports our choice to conduct

individual interviews at t1 to respect their adjusting to the recent diagnosis. Secondly, we

could have addressed dropout at t2 by inviting those who declined to participate in focus

groups for an interview, circumventing possible selection bias in the focus groups. However,

characteristics such as age, gender, and treatment progress were comparable between

participants and nonparticipants. Thirdly, the different interview methods could have

influenced change over time. Furthermore, the outcome prioritizing in the focus groups

enabled us to elicit the breath of views, but ultimate priority setting needs further study using

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appropriate instruments, like questionnaires (48). Lastly, participants were not interviewed at

treatment initiation for practical reasons, and because of their immature disease perspective

early on.

The present study is innovative in two ways. Firstly, it was explicitly oriented toward ERA, a

disease period with specific dynamics. Secondly, interviewing the same participants at

multiple times allowed us to scrutinize patient-preferred outcomes at a disease stage when

opinions could change. Trustworthiness of our findings was increased in five ways. Firstly,

our research team included two trained patient researchers, who provided unique insight on

methodological reflection, interview guide development, data analysis, and manuscript

revision. Secondly, interdisciplinary team discussions gave us a broad view of the data.

Thirdly, inductive analysis ensured that outcomes emerged from participants’ stories.

Fourthly, trial participants shared characteristics with a typical ERA population and were

treated using the same protocol, supporting data transferability. Finally, purposive sampling

aided diversified information gathering.

We conclude that the core patient-preferred outcome of patients with ERA is the urge to

return to a normal life. Over the ERA course, this search for normality involved different

levels: aspects of disease control, physical and mental aspects, and aspects of participation.

There were nuanced changes, but with pain relief remaining prominent. Our study provides

evidence crucial for designing more targeted and holistic therapeutic interventions in ERA.

Additional prospective quantitative research is needed to fully understand the potential of

including patient-preferred outcomes in ERA management.

Acknowledgements

This article would not have been possible without the cooperation of the patients and their

health-care professionals. We also thank the CareRA research team.

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*Patients included in the CareRA trial were eligible for an individual interview if this could be scheduled

within four to six months after start of the initial treatment (t1). t2: at least one year after start of the

initial treatment for early rheumatoid arthritis.

Figure 1. Flow chart of participant recruitment for individual interviews and focus groups

Patients considered ineligible by rheumatologist: N=5

Non-native Dutch speaker: N=1

Loss to follow-up: N=2

Unknown reason: N=2

Patients excluded because rheumatologist declined to participate: N=3

Patients excluded because rheumatologist invited them too late: N=5

Patients declining to participate in an individual interview: N=2

Patients not invited after consideration of data richness: N=5

Participants interviewed individually at t1: N=26

(Treating rheumatologists: n=11)

Participants declining to participate in a focus group: N=7

Participants reconsidering their participation because of scheduling conflicts: N=4

Distance to focus group too far: N= 2

Personal reasons: N=2

Participants interviewed in a focus group at t2: N=14


Participants interviewed individually at t1 and invited to participate in a focus group at t2: N= 25


Participant excluded because treatment discontinued: N=1

Patients enrolled in CareRA trial on January 1, 2012: N=262


Patients eligible* for individual interview at t1: N=46


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Figure 2. Overview of patient-preferred outcomes in early rheumatoid arthritis

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Table 1. Participant demographic, clinical, and self-reported health characteristics grouped

by interview time (t1 or t2)

Participant characteristics Individual

interviews at

t1 (n=26)

Focus groups

at t2 (n=14*)

Median (range) age in years 55 (22-68) 57 (23-66)

Median (range) general health scores

(VAS)†

24 (0-64) 38 (0-80)

Median (range) pain scores (VAS)† 22 (0-65) 37 (0-80)

Median (range) fatigue scores (VAS)† 29 (0-64) 37 (0-80)

Women, n (%) 18 (69) 9 (64)

Site of recruitment, n (%)

Private practice 9 (36) 3 (21)

General hospital 8 (28) 5 (36)

Academic hospital 9 (36) 6 (43)

Geographic location of site, n (%)

Antwerp 1 (4) 0 (0)

Limburg 9 (35) 4 (29)

Flemish Brabant

East Flanders

West Flanders

12 (45)

1 (4)

3 (12)

8 (57)

1 (7)

1 (7)

Treatment progress, n (%)

Patient decided to discontinue

treatment

1 (4) -

Treatment failures‡ in the first year 4 (15) 3 (21)

Treatment failures‡ after one year 1 (4) 1 (7)

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Treatment responders 20 (77) 10 (72)

*Out of the initial 26 participants interviewed at t1, 14 agreed to participate in a

focus group conducted at t2.

†Position marked on a 100-mm visual analog scale (VAS).

‡A treatment failure in the CareRA trial was defined as an efficacy failure when

failing to reach low disease activity even after two predefined treatment

adjustments or as a safety failure in case of persistent toxicity.

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Table 2. Main interview questions and procedures for the individual interviews and focus

groups

Individual

interviews at t1

Interview questions and procedures

Preparatory phase (5 to 10 minutes)

To set the scene for the interview, participants were asked to

write down as many keywords describing:

- the impact of RA on their life

- which outcomes of their illness and treatment they

considered most important.

Start of the interview

The interviews began by discussing participants’ written answers

to those two questions. Participants were asked to elaborate on

their keywords.

- Can you tell me how RA affects your daily life?

- Which outcomes of your illness and antirheumatic

treatment are important to you at this moment?

Proceeding of the interview

The order of the other interview questions was determined by

the participants’ answers during the interview:

- How has the treatment been working for you so far?

- How do you decide whether or not your treatment is

working?

- What made you decide to start treatment?

- What were your expectations of your antirheumatic

treatment at the start of treatment?

- To what extent do the expectations you had at the start

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of your treatment match your current expectations?

*Three questions were added after the first interviews:

Other patients talked about taking less medication*, returning to

a normal life*, feeling better*. Is this something you recognize?

What do you feel about that?

Probing questions: Could you tell me more about that? Could

you give an example?

End of the individual interview: Is there anything else you

would like to add?

Focus groups

at t2

Round 1: Preparatory phase (5 to 10 minutes)

The moderator introduced the phenomenon of interest, after

which each group member was asked to independently prepare

answers to the question below by writing down as many

keywords as possible. Each answer was written on a separate

Post-it®.

- Which outcomes of your illness and antirheumatic

treatment are important to you at this moment?

Next, participants were asked to try to order their Post-its® on a

vertical scale, from most important (top) to least important

(bottom).

Participants were simultaneously asked to think about the

following questions:

- What important treatment results have already been

achieved?

- At present, is there anything you would like to change or

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improve regarding your disease or treatment?

Round 2-step 1: Round-robin listing

All group members were asked to reveal and clarify, one by one,

their personally preferred outcomes in order of importance.

Meanwhile, the observer wrote these outcomes on a flipchart in

front of the group.

- Who would like to share your personally valued

outcomes with the group, in order of importance?

- Could you please clarify why these outcomes of your

disease and antirheumatic treatment are important to

you?

- Why did you designate that specific outcome to be the

most important?

- Is there anything else you would like to add?

Round 2-step 2: Developing a group list of patient-preferred

outcomes

The group was asked to generate a consensus list by reviewing

and merging all recorded outcomes and agreeing on the name

and properties of each outcome on the list.

- Could any of the individual expectations be grouped?

- Who would like to suggest a name and meaning for this

outcome?

- Do you think all the important outcomes are mentioned

on the group list? Is there anything else you would like to

add?

Round 2-step 3: Eliciting personal preferred outcomes

Starting from the consensus list of patient-preferred outcomes

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that resulted in step 2, each group member was asked to

independently try to select his or her five top outcomes from this

list, using the Post-it® ordering scheme.

Round 2-step 4: Eliciting preferred outcomes in the actual

stage of RA

The group was then asked to discuss a collective top 5

outcomes and to consider influencing factors.

- Looking at the group list, what outcome would you order

as most important?

- What outcome would you order secondZfifth?

- Can you tell us why this outcome is either important to

you or not?

End of round 2: That is it for the second round. Is there

anything else to add?

Round 3: Exploring the view of participants on the evolution

of their patient-preferred outcomes over the past year

The focus groups ended by exploring the participants’ views on

potential changes in personally preferred outcomes over time:

During the individual interview of last year, you were asked for

your preferred illness and treatment outcomes. In the meantime,

you have gained more experience with your disease and

treatment and the critical disease stage has passed.

- Do you feel that other results are now more important to

you than the ones you identified at the start or during

your interview last year?

- Could you explain why this has or has not changed?

- Are there outcomes that are now more, less or no longer

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important to you?

- Why do you think that these are now more or less

important than a year ago, or are no longer important?

What may have caused this change in importance?

- Do you have an example of an outcome that has

changed in importance compared to that outcome in the

early disease stage? Why do you think this has

changed? Could you clarify this in more detail?

- In general you mention (more or less) similar/different

outcomes of importance compared to last year (in the

early disease stage). What is your opinion on this

observation?

End of round 3: This is the end of the third round. Is there

anything else to add?

Probing questions: Is this outcome also important or not

important to other group members? Are there any suggestions

from other group members? Is there anyone who has a different

opinion on the matter? Is it difficult for you to share your opinion

on this? Does everyone agree? Who agrees or disagrees and

why? Who would like to add something?

End of the focus group

- What is your general conclusion about today’s focus

group on preferred and important outcomes of disease

and treatment in the actual disease stage?

- To summarize, you talked about [Z].

- Do you agree with this summary of today’s focus group?

t1: Four to six months after start of the initial treatment for early rheumatoid arthritis.

t2: At least one year after start of the initial treatment for early rheumatoid arthritis.

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Table 3. Example of guiding questions based on Saldaña’s method for analyzing longitudinal

data

Set of questions Example questions

Framing questions What contextual factors appear to influence

and affect change in participants’ preferred

outcomes through time?

Descriptive questions What increases or emerges through time?

Analytic and interpretive questions Which changes interrelated through time?

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Table 4. Quotes of participants that illustrate the outcomes preferred by patients with early

rheumatoid arthritis

Return to being normal

“No more pain, just functioning normally again. Like everybody else, just being a

healthy person.” (t1, woman, 29 years old)

“Yet, I think the expectation is ‘being able to do what you could do before’.

(t2, man, 60 years old)

Yeah, that you are still capable of doing things like you were used to.”

(t2, man, 57 years old)

Aspects of disease control

Proof of disease control “If I can see it in my blood that my inflammation goes down

well yesZ there is proof.” (t1, woman, 40 years old )

Prevention or stabilization

of joint damage

“My grandmother also had RA, but her fingers got deformed,

so I thought the faster I could start treatment, the better." (t1,

woman, 29 years old)

Less medication “Medication will always be there. We can’t go on without

those meds.” (t2, man, 51 years old)

Physical aspects

Relief of pain and other

physical symptoms

“Yes in the first instance the pain of course and afterwards the

rest will follow automatically I think.” (t1, woman, 31 years old)

“I am very happy that the pain has gone. That rigid feeling

and so onZwe have to accept it I guess, if that’s all.” (t2,


“Yeah, being tired, I take it into account, so I can go to sleep.”

(t2, man, 51 years old) “Indeed, you can solve it by going to

bed earlier.” (t2, woman, 54 years old)

Improved joint function and “Walking, taking stairs, opening a bottle, tying your

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mobility shoelacesZthese are things every normal person is able to

do, no?” (t2, man, 57 years old)

Limited side effects “I do not expect that all these side effects will disappear, but

there are a number of them that are bothering me and I hope

those improve.” (t2, woman, 60 years old)

Improved sleep “If you can’t sleep no more [because of the pain], then you are

prepared to take the medication again.” (t2, woman, 56 years

old)

Aspects of participation

Performing activities of

daily living

“I do my work independently. Washing, ironing, I do it all back

again myself.” (t1, woman, 65 years old)

“To keep functioning in daily activities, I do find it essential.”

(t2, woman, 54 years old)

Engaging in work and/or

leisure

“If you like to work in the garden, you can do that again. If you

like to bike, you can bike again and if you like to jog, you can

jog again. Things like that.” (t2, man, 57 years old)

“I love doing my job. The rheumatologist said at the start of

treatment: ‘We are going to make sure that you can keep

doing your job.’” (t1, woman, 31 years old)

Fulfilling family, social,

and/or societal roles

“Being able to keep up with the others. Being an ordinary

grandmother.” (t2, woman, 63 years old )

Vitality “That you can get up in the morning and say, ‘I’m going to do

this and that’, and then really being able to realize these plans

and afterwards being energetic enough to do other things.” (t1,


Mental aspects

Emotional well-being “Yes, if you are always crying from pain, or if you are so

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depressed or not being able to sleep and always cryingZ

Now I’m able to laugh and to sing again, and when people are

asking me, ‘How are you?’, you can say, ‘I’m fine!’.” (t1,


“Calmer, being more at ease. How do I have to explain it. In

the beginning you want to be cured, you want it [the disease]

to be gone, to take your medication and it is gone. Now you

know that it’s not going to pass.” (t2, man, 51 years old)

Self and identity “Yeah, you knowZfear that you do not want to give in when

being away in the group. It may sound dramatic but mentally it

wears you down. I’m feeling like a burden to others.” (t1,


“And when I had to drag myself on and on, I thought to myself

‘that’s not me’.” (t1, woman, 49 years old)

Life enjoyment “Just want to enjoy things again, can I say that? I don’t want

to think constantly about the pain, so I can walk again with my

boyfriend and my dog, so I can go for a drink again with

friends or have a nice cozy dinner. Yeah those sorts of

things.” (t1, woman, 31 years old)

Not feeling ill “I am 40 years old and I may not feel like this [ill]. I should be

able to walk normally and not have pain in my foot or knee.

Just not thinking that there is something wrong.” (t1, woman,

40 years old)

Changes over time: “I think a part [of the outcomes] that were mentioned as a priority last

year, are possibly already fulfilled by the tapering of the medication etc., and your reaction is

already better to the medication, that’s why you feel better. So this part is already achieved,

and now you can focus more on the functioning.” (t2, woman, 54 years old)

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