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

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.Electronic supplementary materialThe online version of this article (doi:10.1186/s12969-017-0157-1) contains supplementary material, which is available to authorized users.

The Systemic Juvenile Idiopathic Arthritis Cohort of the Childhood Arthritis and Rheumatology Research Alliance Registry: 2010–2013

“…Demographic features of the CARRA sJIA cohort are similar to previously described smaller cohorts in age at diagnosis, sex, and time to diagnosis 20,21,22 . It will be interesting to compare these Legacy Registry data with the new CARRA Registry data to see whether IL-1 and IL-6 inhibitor use has increased even further over time because more data support early aggressive treatment with these agents 9,26 . Of note, only 7% had current ACR functional classes III or IV, and median CHAQ was 0.1 (IQR 0.0-0.6) at EV, despite 60.4% reporting ACR class III or IV in the past.…”

“…An intriguing hypothesis suggested by several case series of children with recent onset sJIA is that earlier treatment with effective biologic agents may lead to more rapid suppression of disease activity, favorably altering the disease course and reducing steroid exposure 9,26,27 . Longterm followup studies in children with sJIA in the 1993-2002 decade (before the current biologic era) had shown improve-ments in functional outcomes 23,24,28,29,30 and mortality compared with studies in earlier decades 31,32,33,34 .…”

“…Medication choices may also be affected by regional or site preferences 26 . The majority of patients were enrolled in years 1 and 2 (73.4% of cohort), and it is possible that patients enrolled in years 3 and 4 were more likely to be recently diagnosed, which may have affected physician treatment choice.…”

Versorgung von Kindern, Jugendlichen und jungen Erwachsenen mit juveniler idiopathischer Arthritis

“…Ähnliche Initiativen gibt es auch in anderen Ländern, z. B. die CARRA-Initiative in den USA [8,25,26]. Diese bieten die Möglichkeit der weiteren Optimierung der Versorgung von JIA-Patienten.…”

Protokolle zur Klassifikation, Überwachung und Therapie in der Kinderrheumatologie (PRO-KIND): Enthesitis-assoziierte Arthritis

“…Protokolle zur Vereinheitlichung der Diagnostik und Therapie bei juvenilen rheumatischen Erkrankungen wurden unter anderem auch von der CARRA (Childhood Arthritis Rheumatology Research Alliance)-Gruppe erstellt (7)(8)(9)(10). Für die EAA liegen noch keine entsprechenden Protokolle, aber eine retrospektive vergleichende Effektivitätsanalyse vor (11).…”