PT  - JOURNAL ARTICLE
AU  - Suzanne C.  Li
AU  - Kathryn S.  Torok
AU  - C. Egla  Rabinovich
AU  - Fatma Dedeoglu
AU  - Mara L.  Becker
AU  - Polly J.  Ferguson
AU  - Sandy D.  Hong
AU  - Maria F.  Ibarra
AU  - Katie Stewart
AU  - Elena Pope
AU  - Gloria C.  Higgins
AU  - Ronald M.  Laxer
AU  - Thomas  Mason  II
AU  - Robert C.  Fuhlbrigge
AU  - Tracy Andrews
TI  - Initial Results from a Pilot Comparative Effectiveness Study of 3 Methotrexate-based Consensus Treatment Plans for Juvenile Localized Scleroderma
AID  - 10.3899/jrheum.190311
DP  - 2019 Oct 15
TA  - The Journal of Rheumatology
PG  - jrheum.190311
4099  - http://www.jrheum.org/content/early/2020/05/09/jrheum.190311.short
4100  - http://www.jrheum.org/content/early/2020/05/09/jrheum.190311.full
AB  - Objective To perform a comparative effectiveness feasibility study in juvenile localized scleroderma (LS), using standardized treatment regimens (consensus treatment plans; CTP). Methods A prospective, multicenter 1-year pilot observational cohort study was performed by Childhood Arthritis and Rheumatology Research Alliance (CARRA) LS workgroup members. Patients with active, moderate to severe juvenile LS were treated with one of 3 CTP: methotrexate alone, or in combination with intravenous (30 mg/kg/dose for 3 mos) or oral corticosteroids (2 mg/kg/ day tapered by 48 weeks). Results Fifty patients, with demographics typical for juvenile LS, were enrolled, and 44 (88%) completed the study. Most had extracutaneous involvement. Patients improved in all 3 CTP, 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 3 CTP. 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. Response differences were associated with baseline disease activity level, LS subtype, skin disease extent, and extracutaneous involvement. Conclusion This study demonstrates the feasibility of conducting juvenile LS comparative effectiveness studies. The CTP were found to be safe, effective, and tolerable. Our assessments performed well. Because 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.