Methotrexate and corticosteroid therapy for pediatric localized scleroderma

• Published in: The Journal of pediatrics 2000-01, Vol.136 (1), p.91-95
• Main Authors: Uziel, Yosef, Feldman, Brian M, Krafchik, Bernice R, Yeung, Rae S.M, Laxer, Ronald M
• Format: Article
• Language: English
• Subjects: Administration, Oral; Anti-Inflammatory Agents - administration & dosage; Anti-Inflammatory Agents - therapeutic use; Biological and medical sciences; Child; Child, Preschool; Confidence Intervals; Dermatologic Agents - administration & dosage; Dermatologic Agents - adverse effects; Dermatologic Agents - therapeutic use; Drug Therapy, Combination; Female; Follow-Up Studies; Glucocorticoids - administration & dosage; Glucocorticoids - therapeutic use; Humans; Immunosuppressive Agents - administration & dosage; Immunosuppressive Agents - adverse effects; Immunosuppressive Agents - therapeutic use; Injections, Intravenous; Leukopenia - chemically induced; Male; Medical sciences; Methotrexate - administration & dosage; Methotrexate - adverse effects; Methotrexate - therapeutic use; Methylprednisolone - administration & dosage; Methylprednisolone - therapeutic use; Pharmacology. Drug treatments; Placebos; Recurrence; Remission Induction; Scleroderma, Localized - drug therapy; Skin, nail, hair, dermoskeleton
• ISSN: 0022-3476; 1097-6833
• DOI: 10.1016/S0022-3476(00)90056-8

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 esponse 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. Conclusions: 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.