PRELIMINARY EVIDENCE FOR CYCLOSPORINE-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-t wo patients (17 with JRA, 5 with JDMS) with refractory disease were st udied retrospectively. All received CyA at a mean dose of 3.2 mg/k/day over a mean period of 16 mo (range 6-42). All other medications excep t nonsteroidal antiinflammatory drugs, prednisone, and hydroxychloroqu ine were discontinued. In addition, 16/22 patients received concomitan t MTX. Results. Improvements in laboratory variables, joint counts, jo int swelling, and morning stiffness were observed in most of the child ren with JRA. Muscle strength increased and muscle enzyme levels decre ased in the patients with JDMS. CyA treatment permitted prednisone to be discontinued in 5/20 and reduced by greater than 50% in 10/20 patie nts. There was no evidence of hepatic or bone marrow toxicity or lymph oproliferative disease. Serum creatinine increased in 13/22 patients, but the actual values all remained within normal limits. Conclusion. C yA may be an effective agent in the treatment of refractory JRA and JD MS and concomitant MTX seems to be well tolerated. These pulmonary dat a also suggest that combined CyA/MTX therapy may be associated with fu rther improvement in clinical outcome.