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Developing comparative effectiveness studies for a rare, understudied pediatric disease: lessons learned from the
Suzanne C Li1,2, Robert C Fuhlbrigge3, Ronald M Laxer4
1Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Imus PC337, 30 Prospect Ave, Hackensack, NJ, 07061, USA. suzanne.li@hackensackmeridian.org.
Pediatric Rheumatology Online Journal
|July 17, 2019
Summary
This pilot study shows that comparative effectiveness research for juvenile localized scleroderma (jLS) is feasible. Multi-center collaboration and standardized protocols are key to studying rare pediatric diseases.
Area of Science:
- Rheumatology
- Pediatrics
- Clinical Research
Background:
- Limited evidence exists for optimal juvenile localized scleroderma (jLS) therapy.
- A pilot comparative effectiveness study was designed to address this gap.
- The study also evaluated strategies for rare pediatric disease research.
Purpose of the Study:
- To assess the feasibility of conducting a comparative effectiveness study for jLS.
- To identify effective strategies for implementing research in rare pediatric diseases.
- To evaluate standardized methotrexate-based treatment regimens for jLS.
Main Methods:
- Prospective, multi-center, observational cohort study of 50 jLS patients.
- Patients received one of three standardized methotrexate-based consensus treatment plans (CTPs).
- Study conducted by the Childhood Arthritis and Rheumatology Research Alliance (CARRA) from 2012-2015, including a bio-banking sub-study.
Main Results:
- Achieved target enrollment of 50 jLS patients across 10 sites within 26 months.
- Eligibility criteria effectively captured appropriate patients for treatment studies.
- High study completion rate (88%) and significant bio-banking participation (68%) were observed.
Conclusions:
- The approach is feasible for comparative effectiveness research in rare pediatric diseases.
- Multi-center collaboration among dedicated investigators is crucial for success.
- Sufficient investigator numbers and streamlined study management facilitate rare disease research.
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