Methotrexate and corticosteroid therapy for pediatric localized scleroderma

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 gir ls, 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, ail 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. (J Pediatr 2000;136:91-5).