Pilot study comparing the Childhood Arthritis & Rheumatology Research Alliance (CARRA) systemic Juvenile Idiopathic Arthritis Consensus Treatment Plans.

• Type of Material: Article
• Authors: Yukiko Kimura, Hackensack University; Sriharsha Grevich, Seattle Children’s Hospital; Timothy Beukelman, University of Alabama at Birmingham; Esi Morgan, Cincinnati Children’s Hospital Medical Center; Peter A. Nigrovic, Brigham and Women’s Hospital; Kelly Mieszkalski, Childhood Arthritis & Rheumatology Research Alliance; T Brent Graham, Vanderbilt University; Maria Ibarra, Children’s Mercy Hospital; Norman Ilowite, Children’s Hospital at Montefiore; Marisa Klein Klein-Gitelman, Ann & Robert H. Lurie Children’s Hospital of Chicago; Karen Onel, University of Chicago; Sampath Prahalad, Emory University; Marilynn Punaro, Texas Scottish Rite Hospital; Sarah Ringold, Seattle Children’s Hospital; Dana Toib, St. Christopher’s Hospital; Heather Van Mater, Duke University; Jennifer E. Weiss, Hackensack University; Pamela F. Weiss, Children’s Hospital of Philadelphia; Laura E. Schanberg, Duke University
• Language: English
• Date: 2017-04-11
• Publisher: BioMed Central
• Publication Version: Final Published Version
• Copyright Statement: © The Author(s). 2017
• License: Creative Commons Attribution 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1186/s12969-017-0157-1
• Title of Journal or Parent Work: Pediatric Rheumatology
• ISSN: 1546-0096
• Volume: 15
• Issue: 1
• Start Page: 23
• End Page: 23
• Grant/Funding Information: This work was completed with support from an Arthritis Foundation Innovative Research Grant.; The National Institute of Arthritis and Musculoskeletal and Skin Disease at the National Institutes of Health (NIH 1RC1AR058605-01) supported the development of the consensus treatment plans.; The CARRA Registry was supported by grants from the National Institute of Arthritis and Musculoskeletal and Skin Diseases, Friends of CARRA, the Arthritis Foundation, and the NIH (RC2AR058934).
• Keywords: Pediatric rheumatology; Comparative effectiveness; Registries; Systemic Juvenile Idiopathic Arthritis; Biologic response modifiers; Still’s disease
• Research Categories: Health Sciences, Immunology; Health Sciences, Medicine and Surgery

Abstract

OBJECTIVES: To assess the feasibility of studying the comparative effectiveness of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) consensus treatment plans (CTPs) for systemic Juvenile Idiopathic Arthritis (JIA) using an observational registry. METHODS: Untreated systemic JIA patients enrolled in the CARRA Registry were begun on one of 4 CTPs chosen by the treating physician and patient/family (glucocorticoid [GC] alone; methotrexate [MTX] ± GC; IL1 inhibitor [IL1i] ± GC; IL6 inhibitor [IL6i] ± GC). The primary outcome of clinical inactive disease (CID) without current GC use was assessed at 9 months. TRIAL REGISTRATION: clinicaltrials.gov NCT01697254; first registered 9/28/12 (retrospectively enrolled). RESULTS: Thirty patients were enrolled at 13 sites; eight patients were started on a non-biologic CTP (2 GC, 6 MTX) and 22 patients on a biologic CTP (12 IL1i, 10 IL6i) at disease onset. Demographic and disease features were similar between CTP groups. CTP choice appeared to segregate by site preference. CID off GC was achieved by 37% (11 of 30) including 11/22 (50%) starting a biologic CTP compared to 0/8 starting a non-biologic CTP (p = 0.014). There were four serious adverse events: two infections, one appendicitis and one macrophage activation syndrome. CONCLUSIONS: The CARRA systemic JIA CTP pilot study demonstrated successful implementation of CTPs using the CARRA registry infrastructure. Having demonstrated feasibility, a larger study using CTP response to better determine the relative effectiveness of treatments for new-onset systemic JIA is now underway.