Clinical trial of focal segmental glomerulosclerosis in children and young adults

Debbie S. Gipson, Howard Trachtman, Frederick J. Kaskel, Tom H. Greene, Milena K. Radeva, Jennifer J. Gassman, Marva M. Moxey-Mims, Ronald J. Hogg, Sandra L. Watkins, Richard N. Fine, Susan L. Hogan, John P. Middleton, V. Matti Vehaskari, Patti A. Flynn, Leslie M. Powell, Suzanne M. Vento, June L. McMahan, Norman Siegel, Vivette D. D'Agati, Aaron L. Friedman

Research output: Contribution to journalArticlepeer-review

148 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)868-878
Number of pages11
JournalKidney international
Issue number8
StatePublished - Oct 2 2011

Bibliographical note

Funding Information:
This study was sponsored by the NIH/NIDDK Grants U01- DK063385, DK063490, DK063455, and DK063549. This study was supported by many Clinical and Translational Science Award/NIH-funded institutions for the conduct of study visits, nursing, laboratory, and outpatient research facilities throughout the trial. We express our thanks to all the site coordinators who assisted with patient identification, enrollment, treatment, and follow-up.


  • focal segmental glomerulosclerosis
  • proteinuria
  • randomized controlled trial

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