Efficacy and safety of methotrexate therapy for juvenile rheumatoid arthritis.

BACKGROUND AND PURPOSE Juvenile rheumatoid arthritis (JRA) can result in disability, growth disturbance, and systemic complications. This study investigated the efficacy and adverse effects of oral methotrexate (MTX) therapy in Taiwanese children with JRA. METHODS The medical records of 52 Taiwanese children with JRA treated with oral MTX were retrospectively analyzed. The disease onset was polyarticular in 22 children, oligoarticular in 13, and systemic in 17. The indication for MTX therapy was lack of efficacy of previous drugs, including two or more nonsteroidal anti-inflammatory drugs, in all patients. In addition, three patients had received hydroxychloroquine and one had received sulfasalazine, without improvement. Corticosteroid dependency had developed in 27 patients prior to MTX therapy. The mean initial dose of MTX was 9.1 mg.m-2.wk-1 (range, 5-20 mg.m-2.wk-1), and the mean maximal dose was 10.2 mg.m-2.wk-1 (range, 5-20 mg.m-2.wk-1). The mean duration of treatment was 23 months (range, 6-96 mo), and the mean duration of follow-up was 52 months (range, 10-123 mo) from the start of MTX therapy. RESULTS Thirty-six children (69%) showed clinical improvement and 25 children (48%) achieved clinical remission. The administration of MTX resulted in more than a 50% reduction in required corticosteroid dosage in six children, and complete discontinuation of corticosteroid in 10 children. MTX was discontinued in 18 patients following a mean of 8 months (range, 2-34 mo) of clinical remission. Relapse occurred in nine (50%) of these patients. Thirteen patients (25%) suffered from adverse effects associated with MTX treatment. All of these adverse effects resolved spontaneously, or subsided within 4 weeks following dosage reduction or discontinuation of MTX. CONCLUSIONS Oral MTX therapy is effective and well-tolerated in Taiwanese children with JRA. It can serve as the first choice of second-line therapy in JRA.