Clinical trial of focal segmental glomerulosclerosis in children and young adults.

Journal Article (Journal Article;Multicenter Study)

This NIH-funded multicenter randomized study of focal segmental glomerulosclerosis (FSGS) treatment compared the efficacy of a 12-month course of cyclosporine to a combination of oral pulse dexamethasone and mycophenolate mofetil in children and adults with steroid-resistant primary FSGS. Of the 192 patients enrolled, 138 were randomized to cyclosporine (72) or to mycophenolate/dexamethasone (66). The primary analysis compared the levels of an ordinal variable measuring remission during the first year. The odds ratio (0.59) for achieving at least a partial remission with mycophenolate/dexamethasone compared to cyclosporine was not significant. Partial or complete remission was achieved in 22 mycophenolate/dexamethasone- and 33 cyclosporine-treated patients at 12 months. The main secondary outcome, preservation of remission for 26 weeks following cessation of treatment, was not significantly different between these two therapies. During the entire 78 weeks of study, 8 patients treated with cyclosporine and 7 with mycophenolate/dexamethasone died or developed kidney failure. Thus, our study did not find a difference in rates of proteinuria remission following 12 months of cyclosporine compared to mycophenolate/dexamethasone in patients with steroid-resistant FSGS. However, the small sample size might have prevented detection of a moderate treatment effect.

Full Text

Duke Authors

Cited Authors

  • Gipson, DS; Trachtman, H; Kaskel, FJ; Greene, TH; Radeva, MK; Gassman, JJ; Moxey-Mims, MM; Hogg, RJ; Watkins, SL; Fine, RN; Hogan, SL; Middleton, JP; Vehaskari, VM; Flynn, PA; Powell, LM; Vento, SM; McMahan, JL; Siegel, N; D'Agati, VD; Friedman, AL

Published Date

  • October 2011

Published In

Volume / Issue

  • 80 / 8

Start / End Page

  • 868 - 878

PubMed ID

  • 21734640

Pubmed Central ID

  • PMC3306824

Electronic International Standard Serial Number (EISSN)

  • 1523-1755

Digital Object Identifier (DOI)

  • 10.1038/ki.2011.195


  • eng

Conference Location

  • United States