Y Uziel1, B M Feldman, B R Krafchik, R S Yeung, R M Laxer. 1. Division of Rheumatology and Pediatric Medicine, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
Abstract
INTRODUCTION: Localized scleroderma (LS) can cause permanent disability, and there is no universally accepted effective treatment. Methotrexate (MTX) has been shown to be effective in the treatment of systemic sclerosis. OBJECTIVES: To determine the efficacy and tolerability of MTX and corticosteroid therapy in patients with LS. METHODS:MTX, 0.3 to 0.6 mg/kg per week, was given to 10 patients (6 girls, 4 boys; mean age, 6.8 years; mean disease duration before starting treatment, 4 years) with active LS. In addition, pulse intravenous methylprednisolone, 30 mg/kg for 3 days monthly for 3 months, was given to 9 patients at the initiation of therapy. RESULTS: One patient discontinued taking MTX after a month; the remaining 9 patients responded. The median time to response was 3 months (95% CI, 1.15-4.85). One responder discontinued taking MTX after a year because of leukopenia; the LS worsened within 2 months. In another patient LS flared up after 10 months and responded to an increased dose of MTX and intravenous methylprednisolone. At the last follow-up visit, all patients who continued to receive MTX therapy had inactive skin lesions. CONCLUSION: Treatment with MTX and corticosteroids appears to be effective in the treatment of LS and is generally well tolerated. A placebo-controlled study is necessary to confirm the efficacy of MTX therapy in LS.
RCT Entities:
INTRODUCTION:Localized scleroderma (LS) can cause permanent disability, and there is no universally accepted effective treatment. Methotrexate (MTX) has been shown to be effective in the treatment of systemic sclerosis. OBJECTIVES: To determine the efficacy and tolerability of MTX and corticosteroid therapy in patients with LS. METHODS:MTX, 0.3 to 0.6 mg/kg per week, was given to 10 patients (6 girls, 4 boys; mean age, 6.8 years; mean disease duration before starting treatment, 4 years) with active LS. In addition, pulse intravenous methylprednisolone, 30 mg/kg for 3 days monthly for 3 months, was given to 9patients at the initiation of therapy. RESULTS: One patient discontinued taking MTX after a month; the remaining 9 patients responded. The median time to response was 3 months (95% CI, 1.15-4.85). One responder discontinued taking MTX after a year because of leukopenia; the LS worsened within 2 months. In another patient LS flared up after 10 months and responded to an increased dose of MTX and intravenous methylprednisolone. At the last follow-up visit, all patients who continued to receive MTX therapy had inactive skin lesions. CONCLUSION: Treatment with MTX and corticosteroids appears to be effective in the treatment of LS and is generally well tolerated. A placebo-controlled study is necessary to confirm the efficacy of MTX therapy in LS.
Authors: Suzanne C Li; Kathryn S Torok; Elena Pope; Fatma Dedeoglu; Sandy Hong; Heidi T Jacobe; C Egla Rabinovich; Ronald M Laxer; Gloria C Higgins; Polly J Ferguson; Andrew Lasky; Kevin Baszis; Mara Becker; Sarah Campillo; Victoria Cartwright; Michael Cidon; Christi J Inman; Rita Jerath; Kathleen M O'Neil; Sheetal Vora; Andrew Zeft; Carol A Wallace; Norman T Ilowite; Robert C Fuhlbrigge Journal: Arthritis Care Res (Hoboken) Date: 2012-08 Impact factor: 4.794