Preliminary evidence for cyclosporin A as an alternative in the treatment of recalcitrant juvenile rheumatoid arthritis and juvenile dermatomyositis.

OBJECTIVE: To evaluate the safety and efficacy of cyclosporin A (CyA) with and without methotrexate (MTX) in refractory juvenile rheumatoid arthritis (JRA) and juvenile dermatomyositis (JDMS).

METHODS: Twenty-two patients (17 with JRA, 5 with JDMS) with refractory disease were studied retrospectively. All received CyA at a mean dose of 3.2 mg/kg/day over a mean period of 16 mo (range 6-42). All other medications except nonsteroidal antiinflammatory drugs, prednisone, and hydroxychloroquine were discontinued. In addition, 16/22 patients received concomitant MTX.

RESULTS: Improvements in laboratory variables, joint counts, joint swelling, and morning stiffness were observed in most of the children with JRA. Muscle strength increased and muscle enzyme levels decreased in the patients with JDMS. CyA treatment permitted prednisone to be discontinued in 5/20 and reduced by greater than 50% in 10/20 patients. There was no evidence of hepatic or bone marrow toxicity or lymphoproliferative disease. Serum creatinine increased in 13/22 patients, but the actual values all remained within normal limits.

CONCLUSION: CyA may be an effective agent in the treatment of refractory JRA and JDMS and concomitant MTX seems to be well tolerated. These preliminary data also suggest that combined CyA/MTX therapy may be associated with further improvement in clinical outcome.