Abstract
BackgroundHerein we describe the history, design, and rationale of the new Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry and present the characteristics of patients with juvenile idiopathic arthritis (JIA) enrolled in the first 12 months of operation.MethodsThe CARRA Registry began prospectively collecting data in the United States and Canada in July 2015 to evaluate the safety of therapeutic agents in persons with childhood-onset rheumatic disease, initially restricted to JIA. Secondary objectives include the evaluation of disease outcomes and their associations with medication use and other factors. Data are collected every 6 months and include clinical assessments, detailed medication use, patient-reported outcomes, and safety events. Follow-up is planned for at least 10 years for each participant and is facilitated by a telephone call center.ResultsAs of July 2016, 1192 patients with JIA were enrolled in the CARRA Registry at 49 clinical sites. At enrollment, their median age was 12.4 years old and median disease duration was 2.6 years. Owing to preferential enrollment, patients with systemic JIA (13%) and with a polyarticular course (75%) were over-represented compared to patients in typical clinical practice. Approximately 49% were currently using biologic agents and ever use of oral glucocorticoids was common (47%). The CARRA Registry provides safety surveillance data to pharmaceutical companies to satisfy their regulatory requirements, and several independently-funded sub-studies that use the Registry infrastructure are underway.ConclusionThe new CARRA Registry successfully enrolled nearly 1200 participants with JIA in the first 12 months of its operation. Sustainable funding has been secured from multiple sources. The CARRA Registry may serve as a model for the study of other uncommon diseases.
Highlights
We describe the history, design, and rationale of the new Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry and present the characteristics of patients with juvenile idiopathic arthritis (JIA) enrolled in the first 12 months of operation
During its operation from 2010 through 2014, the CARRA Legacy Registry successfully enrolled the largest number of prospectively followed pediatric rheumatology patients to date: 9,587 participants including 6,607 with JIA, 1,217 with childhood-onset systemic lupus erythematosus (cSLE), and 688 with juvenile dermatomyositis (JDM)
The primary objective of the CARRA Registry is to prospectively collect data essential to evaluate the safety of therapeutic agents in children, adolescents, and young adults with childhood-onset rheumatic diseases
Summary
We describe the history, design, and rationale of the new Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry and present the characteristics of patients with juvenile idiopathic arthritis (JIA) enrolled in the first 12 months of operation. Even though JIA is the most common pediatric rheumatologic condition with a prevalence of approximately 1 per 1000 children, the current understanding of its pathogenesis, natural history, and long-term outcomes is limited [2]. Over the last 15 years, the adoption of highly effective biologic therapeutic agents has dramatically changed the treatment and expected outcomes in JIA. Despite the widespread use of biologic agents, important safety questions remain unanswered, regarding potential adverse effects that are rare or have a long latency period. Safety information about more recently approved biologic agents in children remains very limited. There is little published high-quality evidence to guide pediatric rheumatologists in the management of childhood-onset systemic lupus erythematosus (cSLE), juvenile dermatomyositis (JDM), localized scleroderma, and other less common conditions
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