The Childhood Arthritis and Rheumatology Research Alliance Consensus Treatment Plans

Abstract

The pediatric rheumatic diseases are a heterogeneous group of rare diseases, posing a number of challenges for the use of traditional clinical and translational research methods. Innovative comparative effectiveness approaches are needed to efficiently study treatment strategies and disease outcomes. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed the consensus treatment plan (CTP) approach as a comparative effectiveness tool for research in pediatric rheumatology. CTPs are treatment strategies, developed by consensus methods among CARRA members, intended to reduce variation in treatment approaches, standardize outcome measurements, and allow for comparison of the effectiveness of different approaches with the goal of improving disease outcomes. To date, CTPs have been published for 8 different diseases and disease manifestations. The approach has been successfully piloted for juvenile localized scleroderma, systemic juvenile idiopathic arthritis (JIA), polyarticular JIA, dermatomyositis, and lupus nephritis. Large-scale studies are underway for systemic JIA and polyarticular JIA, with the CARRA patient registry serving as the data collection platform. These studies have been designed with stakeholder involvement, including active input from CARRA providers, patients, and parents, with the goal of increasing feasibility and ensuring the relevance of the outcomes. These studies include ancillary biologic specimen collection intended to support additional translational and mechanistic studies. Data from these ongoing CTP studies will provide more information on the ability of this approach to identify effective treatment strategies and improve outcomes in the pediatric rheumatic diseases.

Efficacy and safety of tocilizumab in a real-life observational cohort of patients with polyarticular juvenile idiopathic arthritis

“…Randomized controlled trials remain the gold standard for establishing the efficacy and short-term safety of new therapeutic agents. However, such studies have strict criteria of patient selection with regard to disease duration and disease severity; in addition, the withdrawal design used in the JIA biological agent studies can overestimate the effect of the trial agent [14,15]. Thus, the results of randomized controlled trials are hard to generalize to real-life clinical practice.…”

Timing matters: real-world effectiveness of early combination of biologic and conventional synthetic disease-modifying antirheumatic drugs for treating newly diagnosed polyarticular course juvenile idiopathic arthritis

“…Although not statistically significantly different, the 6.35 (95% CI −5.89 to 18.58) treatment effect in PedsQL scores at 12 months was greater than the established minimal clinically important difference of 4.5 16. The ongoing prospective cohort study evaluating the effectiveness of consensus treatment plans for pcJIA is expected to bring more clarity 24…”

Efectividad de la terapia Watsu en pacientes con artritis idiopática juvenil. Un ensayo clínico controlado paralelo, aleatorio y simple ciego

“…Por último, consideramos que realizar un diseño de estudio de alto nivel de evidencia en una población reumatológica pediátrica como la AIJ, es un gran desafío, dada la complejidad para cumplir los requerimientos metodológicos en términos de factibilidad y generalización de resultados 34 . Esto debido a que la AIJ aún es considerada una enfermedad rara dada su baja prevalencia 7 y al énfasis de estudiar mediante este tipo de diseño la efectividad de las terapias farmacológicas principalmente.…”