OBJECTIVES: Juvenile localized scleroderma (JLS) is a chronic progressive fibrotic process of the skin causing permanent disability and aesthetic damage. We aimed to assess the safety and efficacy of MTX in patients with JLS. METHODS: Patients with active JLS were randomized (2:1) to double-blinded oral MTX, 15 mg/m(2) (max 20 mg) or placebo, once a week, for 12 months or until treatment failure. Both groups received oral prednisone (1 mg/Kg/day, max 50 mg) for the first three months. A target lesion was evaluated clinically, with infrared thermography and using a computerized scoring system with skin score rate (SSR) evaluation. Response to treatment was defined as: no new lesions; SSR<1; decrease lesion temperature by at least 10% compared to baseline. Treatment failure was defined by new lesions, SSR>1, or increased lesion temperature. All analyses were intention-to-treat. RESULTS: Of 85 screened patients, 70, aged 6-17 years, were randomized, 46 to MTX, 24 to placebo. Mean disease duration was 2.3 years. After an initial response in all patients, disease relapsed in 15 MTX patients (32.6%) and 17 placebo (70.8%) (p<0.005). New lesions appeared in 3 MTX patients (6.5%) vs 4 on placebo (16.7%), mean SSR decreased from 1 to 0.79 with MTX vs 1.1 on placebo, mean target lesion temperature decreased 44.4% vs 12.1%. 26 patients (56.5%) of MTX group and 11 patients (45.8%) of the placebo group presented mild side effects related to treatment. None was severe enough to stop treatment.
Methotrexate in juvenile localized scleroderma: A randomised, double-blind, placebo-controlled trial.
MARTINO, Silvana;
2011-01-01
Abstract
OBJECTIVES: Juvenile localized scleroderma (JLS) is a chronic progressive fibrotic process of the skin causing permanent disability and aesthetic damage. We aimed to assess the safety and efficacy of MTX in patients with JLS. METHODS: Patients with active JLS were randomized (2:1) to double-blinded oral MTX, 15 mg/m(2) (max 20 mg) or placebo, once a week, for 12 months or until treatment failure. Both groups received oral prednisone (1 mg/Kg/day, max 50 mg) for the first three months. A target lesion was evaluated clinically, with infrared thermography and using a computerized scoring system with skin score rate (SSR) evaluation. Response to treatment was defined as: no new lesions; SSR<1; decrease lesion temperature by at least 10% compared to baseline. Treatment failure was defined by new lesions, SSR>1, or increased lesion temperature. All analyses were intention-to-treat. RESULTS: Of 85 screened patients, 70, aged 6-17 years, were randomized, 46 to MTX, 24 to placebo. Mean disease duration was 2.3 years. After an initial response in all patients, disease relapsed in 15 MTX patients (32.6%) and 17 placebo (70.8%) (p<0.005). New lesions appeared in 3 MTX patients (6.5%) vs 4 on placebo (16.7%), mean SSR decreased from 1 to 0.79 with MTX vs 1.1 on placebo, mean target lesion temperature decreased 44.4% vs 12.1%. 26 patients (56.5%) of MTX group and 11 patients (45.8%) of the placebo group presented mild side effects related to treatment. None was severe enough to stop treatment.
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