Methotrexate in Linear Scleroderma: long-term efficacy in 50 children from a single Pediatric Rheumatology Centre

OBJECTIVE: to study disease course and long-term outcome of children with Linear Scleroderma (LiS) treated with methotrexate (MTX) since diagnosis.METHODS: Retrospective and cross-sectional study including consecutive children with LiS treated with MTX for >1 year and with at least 2 years follow-up. Disease course was analyzed by number of relapses and treatment changes. Relapse-free survival (RFS) was examined by Kaplan-Meier analysis comparing patients with LiS and other JLS subtypes. Disease activity and damage were assessed by Localized Scleroderma Cutaneous Assessment Tool (LoSCAT) and thermography.RESULTS: Fifty patients with mean follow-up 7.8 years and MTX treatment duration 3.1 years were included. Sixteen percent did not respond to first course of MTX and 16% had at least one flare. Complete remission was observed in 18.2% patients with 2-5 years follow-up, 80.0% by 10 years and 87.5% by >10 years. No significant difference in RFS between patients with LiS and 17 with other JLS subtypes was observed. Tissue damage was mild in 42% patients, moderate in 32% and severe in 26%. No significant correlation between severity of tissue damage and LiS subtype, disease duration, relapses and remission emerged. Treatment duration was significantly related to disease relapses (p<0.05) and severity of tissue damage (p<0.005).CONCLUSION: Most patients with LiS treated with MTX achieve complete and long-lasting remission. Overall aesthetic and functional sequelae are moderate, probably because tissue damage establishes early and treatment likely stabilizes it. Early diagnosis and MTX treatment as well as long-term monitoring are crucial to improve outcome and promptly identify flares.