A randomized trial of cyclosporine in patients with steroid-resistant focal segmental glomerulosclerosis

Background. A clinical trial of cyclosporine in patients with steroid-resis tant focal segmental glomerulosclerosis (FSGS) was conducted. Despite the f act that it is the most common primary glomerulonephritis to progress to re nal failure, treatment trials have been very limited. Methods. We conducted a randomized controlled trial in 49 cases of steroid- resistant FSGS comparing 26 weeks of cyclosporine treatment plus low-dose p rednisone to placebo plus prednisone. All patients were followed for an ave rage of 200 weeks, and the short- and long-term effects on renal function w ere assessed. Results. Seventy percent of the treatment group versus 4% of the placebo gr oup (P < 0.001) had a partial or complete remission of their proteinuria by 26 weeks. Relapse occurred in 40% of the remitters by 52 weeks and 60% by week 78, but the remainder stayed in remission to the end of the observatio n period. Renal function was better preserved in the cyclosporine group. Th ere was a decrease of 50% in baseline creatinine clearance in 25% of the tr eated group compared with 52% of controls (P < 0.05). This was a reduction in risk of 70% (95% CII 9 to 43) independent of other baseline demographic and laboratory variables. Conclusions. These results suggest that cyclosporine is an effective therap eutic agent in the treatment of steroid-resistant cases of FSGS. Although a high relapse rate does occur, a longterm decrease in proteinuria and prese rvation of filtration function were observed in a significant proportion of treated patients.