Transitional Care for PediatricPatients with Neuromuscular

Diseases: A Health Technology Assessment

Jackie Tran, MDUniversity of Medicine and Dentistry of New

Jersey, USA

HTAi 9th Annual Meeting, BilbaoIntegrated Care for a Patient Centered System

25 June, 2012

Outline

Background Transitional Care Overview Neuromuscular Diseases Overview

Objectives Methods Results

Barriers Recommendations

Conclusions

Transitional Care OverviewBackground

Unique Adult Needs

Physical maturation Size Endocrine management Deformities

Sexual maturation Menstruation Fertility Pregnancy

Psychosocial maturation Identity Experimentation Independence Family/Friends

Intellectual maturation Education Vocation

Reality: Unmet Needs

Increase hospitalization, particularly of ages 18-21

© Pediatrics. 2011 Jul;128(1):5-13. Epub 2011 Jun 27.Adults with chronic health conditions originating in childhood: inpatient experience in children's hospitals.

Reality: Unmet Needs

Non-adherence to medications over a lifetime in renal transplant patients

Decrease clinic attendance and an average of 1.47 health provider changes in diabetes type 1 patients

Decrease mobility in spina bifida patients during transitional years

Increase long-term morbidity and mortality in cystic fibrosis patients

Neuromuscular Diseases Overview

Background

Selected Neuromuscular Diseases

of ChildhoodCongenital

myopathies dystrophic (e.g., Duchenne) myotonic congenital

spinal muscle atrophy congenital myasthenia hereditary motor

sensory neuopathy Friedreich ataxia

Acquired

Guillain-Barré syndrome

myasthenia gravis polymyositis toxic myopathy

Epidemiology

Worldwide prevalence of disabling, inherited neuromuscular diseases: 1 in 3,500

Increased survival rate to adulthoodSome Survival Statistics in the Literature

60% of males born with Duchenne between 1983 – 1987 survived past age 24

More than 33% of Charcot-Marie-Tooth Type 2 patients live past the third decade of life

A patient with Type 2 SMA have survival rate of 68.5% at age 25

Unique Characteristics

Low recognition Apparent functional deficits Infamous progressive/regressive

courses Media portrayal Multi-organ systems involvement Large team investment

Equally Poor Transitional Care

A glaring 60% of 850 people living with muscle disease rate transition from child to adult services as “poor” or

“very poor”

Muscular Dystrophy Campaign 2008 survey

Finding Transitional Solutions

Objectives & Methods

Objectives

Identify systematic and unique barriers to a successful transition of care for adolescents with neuromuscular diseases (NMDs).

Recommend comprehensive strategies to improve the experience for patients, family members, and health providers alike.

Methods

Systematic literature review of publications between January 2000 and December 2011

Informal survey of local clinicians’ opinions

Only the beginning…Results

General Observations

Notable publication increase on transitional care over the past 5 years

Emphasis on the following diagnoses: diabetes, cancer, renal transplant, inflammatory bowel

dz cerebral palsy and spina bifida

Most prolific countries: USA, Canada and UK Honorable mentions: Australia, France, Japan,

Switzerland

All Transitional Care Publications

NMD-Relevant Publications

Number of

Publications

60+ 14

Population description

• generic: “chronic illnesses” & “special health care needs” & “congenital disabilities” & “developmental

disabilities”

• multiple diagnoses-specific publications

• inter-mix with spina bifida & cerebral palsy &

“chronic neurological disorders”

• Duchenne dystrophy

All Transitional Care Publications

NMD-Relevant Publications

Publication

Content

• highlight need for transitional care• clinician & patient survey/commentaries

• anecdote / case reports

• single-institution experience

• general critiques of current transition

processes & models

• review of medical topics on transitional ages

Outcome measure

s

• subjective reports of wellness & satisfaction

• individual programs: admissions & biomarkers

(retrospective & limited to 1 year after transition)

• health expenditure at transitional age as a secondary outcome

Models of Transitional Care

Diagnosis-focused (most common)

pediatric and adult provider collaboration

Medical home-based (least common)

primary care practitioner as coordinator

Adolescent-focused physical, physiological, social issues focused

Transition-based outreach representatives from one facility

General Barriers

Afterthought process Provider availability (absolute & time)

Coordinator responsibility Decision-making capacity Advisors resourcefulness Insurance coverage Transportation / Architectural Medical records Ventilator, dialysis

Emotional Barriers

Patient & Family neglect

abandonment

Adult team burden

unfamiliarity

Peds team attachment

over-confidence

FearAnxietyDistrust

Uncertainty

Unique Barriers &

Opportunities

Assistive technology Malpractice insurance

Insurance / resources for the very ill Association with spina bifida and/or cerebral

palsy

Even more limited provider availability Institutionalized patients Aging care providers Uncertain future

Barriers

Opportunities

Recommendations

Early notification Individualized plan

flexible but definitive date of transition assess cognitive/emotional and functional

status gradual promote of independence

Comprehensive approach patient’s and family members’ input interdisciplinary team

Recommendations

Coordinator designation Patient education

self care and disease process healthcare system navigation

Long-term planning vocation and education living situation

Resources availability

Advanced Topics

Program sustainability Provider training Transition to palliative care Socioeconomic status effects

Conclusions

All children with neuromuscular diseases need a transition plan before entering adulthood

Successful transition requires coordination of services across all providers

Especially for individuals affected by NMDs, the transition plan should incorporate patient’s and family’s long-term goals

Primary References

Abbott, D. Transition to adulthood for young men with Duchenne Muscular Dystrophy. International Journal of Integrated Care, 31 December 2009 - ISSN 1568-415

Centers for Disease Control and Prevention (CDC). Prevalence of Duchenne/Becker muscular dystrophy among males aged 5-24 years - four states, 2007. MMWR Morb Mortal Wkly Rep. 2009 Oct 16;58(40):1119-22.

Strehle EM. Long-term management of children with neuromuscular disorders. J Pediatr (Rio J). 2009 Sep-Oct;85(5):379-84. Tiffreau V, et al. Ann Readapt Med Phys. 2006 Dec;49(9):652-8. Epub 2006 Jun 27. [Transition in health care from youth to

adulthood for disabled people]. Ouyang L, et al. Health care utilization and expenditures for childrenand young adults with muscular dystrophy in a privately insured

population. J Child Neurol. 2008 Aug;23(8):883-8. Epub 2008 Apr 10. Manzur AY, Muntoni F. Diagnosis and new treatments in musculardystrophies. J Neurol Neurosurg Psychiatry. 2009 Jul;80(7):706-

14. Hill ME, Phillips MF. Service provision for adults with long-term disability: a review of services for adults with chronic

neuromuscular conditions in the United Kingdom. Neuromuscul Disord. 2006 Feb;16(2):107-12. Epub 2006 Jan 19. Nomura Y. [Care continuity for patients with myasthenia gravis during transition from childhood to adulthood]. Nihon Rinsho. 2010

Jan;68(1):39-44. Yoshioka M, et al. [Care continuity for patients with myopathy during transition of childhood to adulthood]. Nihon Rinsho. 2010

Jan;68(1):53-6. Minicozzi A. Transition to adult care: another view. Pediatr Nurs. 2000 Jul-Aug;26(4):411-2. Katz JD, et al. Parents' perception of self-advocacy of children with myositis: an anonymous online survey. Pediatr Rheumatol Online

J. 2011 Jun 7;9(1):10. Goodman DM, et al. Adults with chronic health conditions originating in childhood: inpatient experience in children's hospitals.

Pediatrics. 2011 Jul;128(1):5-13. Epub 2011 Jun 27. Rearick E. Enhancing success in transition service coordinators: use of transformational leadership. Prof Case Manag. 2007 Sep-

Oct;12(5):283-7. Parker AE, et al. Analysis of an adult Duchenne muscular dystrophy population. QJM. 2005 Oct;98(10):729-36. Epub 2005 Aug 31.