A MULTICENTER STUDY OF ALTERNATE-DAY PREDNISONE THERAPY IN PATIENTS WITH CYSTIC-FIBROSIS

The purpose of this study was to evaluate the efficacy and safety of a lternate-day prednisone therapy in treating patients with mild-to-mode rate cystic fibrosis during a 4-year period. In 15 North American cyst ic fibrosis centers, we screened 320 patients and enrolled 285 patient s from April 1986 to December 1987. Patients were randomly assigned to take prednisone, 1 mg/kg per dose or 2 mg/kg per dose, or a matching placebo given on alternate days. Lung function, clinical status, hospi talizations, growth, and steroid side effects were monitored, During t he first 24 months the percentage of the predicted forced vital capaci ty was greater in the 1 mg/kg group (p <0.0001) and the 2 mg/kg group (p <0.01) when each was compared with placebo. Patients in the 1 mg/kg group had a higher percentage of predicted forced vital capacity than placebo patients during the entire 48 months (p <0.0025), but only in the group of patients who were colonized with Pseudomonas aeruginosa at baseline. For 48 months, the 1 mg/kg group had a higher percentage of predicted forced expiratory volume in 1 second than patients given placebo (p <0.02). The prednisone-treated groups had a reduction in se rum IgG concentrations (1 mg/kg vs placebo, p <0.007; 2 mg/kg vs place bo, p <0.003). From 6 months onward, height z scores fell in the 2 mg/ kg group compared with those given placebo (p <0.001). For the I mg/kg group, height z scores were lower from 24 months, An excess of abnorm alities in glucose metabolism was seen in the 2 mg/kg group compared w ith the placebo group (p <0.005). Our findings suggest a role for alte rnate-day prednisone therapy at a dose of 1 mg/kg in patients with mil d to moderate cystic fibrosis. The benefit of improved lung function a ppears to outweigh the potential for adverse effects when the treatmen t period is less than 24 months.