BACKGROUND Cyclosporine, a potent immunosuppressive drug, has been suggested to be beneficial in the treatment of some immune-mediated dermatoses, including pemphigus. OBJECTIVE The aim of the present study was to evaluate the efficacy and safety of combined treatment with prednisone and cyclosporine compared with prednisone alone in patients with pemphigus. METHODS Sixteen hospitalized patients with pemphigus vulgaris received combined treatment with cyclosporine and prednisone for 12 months. Cyclosporine and prednisone were given orally at an initial dose of 5 mg/kg/day and 60 to 80 mg/day, respectively. The cyclosporine dose was adjusted to obtain plasma levels of 100 to 150 ng/L. A historical control group was composed of 15 patients with pemphigus who received prednisone at an initial dose of 120 mg/day, which was decreased according to clinical response. The clinical response, serum titer of autoantibodies, and frequency of side effects during a 1-year follow-up period were evaluated and compared. RESULTS The 16 patients in the combined therapy group achieved clinical remission within 25 days or less, a shorter period than for the control group, although the difference was not statistically significant. New blister formation ceased after a mean of 11.1 +/- 7.9 days of onset of treatment in the combined treatment group versus 20.5 +/- 12 days in the control group (p = 0.004). Hospital stay was shorter in the combined treatment group (mean, 32.6 +/- 12.5 days) than in the control group (mean, 50.7 +/- 17.1 days; p = 0.003).(ABSTRACT TRUNCATED AT 250 WORDS)