Abstract
ObjectivesTo 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.MethodsUntreated 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).ResultsThirty 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.ConclusionsThe 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.
Highlights
Systemic Juvenile Idiopathic Arthritis (JIA) is a rare childhood inflammatory disease associated with significant morbidity, and characterized by arthritis accompanied by high spiking fevers, plus additional features such as rash, generalized lymphadenopathy, hepatosplenomegaly, and serositis [1]
Thirty untreated, mostly newly diagnosed systemic JIA patients were enrolled into the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry at 13 sites and started on a CARRA systemic JIA Consensusderived standardized Treatment Plans (CTPs)
There were 4 serious adverse events (SAEs)’s: 2 that resulted in hospitalization for intravenous antimicrobial therapy, 1 hospitalization for Discussion This pilot study assessed the feasibility of using standardized CARRA CTPs for untreated systemic JIA, 90% of whom had disease onset less than 4 months prior to treatment, to evaluate the comparative effectiveness of commonly used treatment approaches
Summary
Systemic Juvenile Idiopathic Arthritis (JIA) is a rare childhood inflammatory disease associated with significant morbidity, and characterized by arthritis accompanied by high spiking fevers, plus additional features such as rash, generalized lymphadenopathy, hepatosplenomegaly, and serositis [1]. Comparative effectiveness research (CER) studies in an observational, routine care setting are a more practical approach to examine which treatments are effective and most appropriate for an individual child [15, 16] In such a study, physician choice (together with patient/caregiver preferences) would be the primary determining factor for treatment selection. The development of Consensusderived standardized Treatment Plans (CTPs) for systemic JIA funded by a National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) Challenge Grant (1RC1AR058605-01) to the Childhood Arthritis and Rheumatology Research Alliance (CARRA) was a key step in this process and in improving outcomes for patients with systemic JIA [17, 18]. The methods by which the CARRA CTPs were developed using CARRA-wide surveys to identify the current most commonly used treatments for systemic JIA and the standardization process using consensus methodology are outlined in a previously published article [17]
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