Initial Results from a Pilot Comparative Effectiveness Study of Three Methotrexate-Based Consensus Treatment Plans for Juvenile Localized Scleroderma
Abstract
Objective To perform a comparative effectiveness feasibility study in juvenile localized scleroderma (jLS), using standardized treatment regimens (consensus treatment plans, CTPs).
Methods A prospective, multi-center 1-year pilot observational cohort study was performed by Childhood Arthritis and Rheumatology Research Alliance (CARRA) localized scleroderma workgroup members. Active, moderate to severe jLS patients were treated with one of three CTPs: methotrexate alone, or in combination with intravenous (30 mg/kg/dose for 3 months) or oral corticosteroids (2 mg/kg/day tapered off by 48 weeks).
Results Fifty patients, with demographics typical for jLS, were enrolled, and 44 (88%) completed the study. Most had extracutaneous involvement. Patients improved in all three CTPs, with >75% having a major or moderate level of improvement compared to baseline. Damage accrued in some patients. Major deviations from prescribed regimen resulted from medication intolerance (n = 6, 14%) or treatment failure (n = 11, 25%); failures occurred in all three CTPs. Significant responses to treatment were demonstrated by LS skin scoring measures and overall physician assessments, with differences in response level identified in some patient subsets. Baseline disease activity level, LS subtype, skin disease extent, and extracutaneous involvement were associated with response differences.
Conclusion This study demonstrates the feasibility of conducting jLS comparative effectiveness studies. The CTPs were found to be safe, effective, and tolerable. Our assessments performed well. As damage is common and may progress despite effective control of activity, we recommend initial treatment efficacy be evaluated primarily by activity measures. Potential confounders for response were identified that warrant further study.