title: Clinical trial of focal segmental glomerulosclerosis in children and young adults.
abstract: 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.
authors: Division of Nephrology, Department of Pediatrics & Communicable Diseases, University of Michigan, Ann Arbor, Michigan, USA. dgipson@umich.edu
authors: Debbie S
authors: DS
authors: Gipson
authors: Howard
authors: H
authors: Trachtman
authors: Frederick J
authors: FJ
authors: Kaskel
authors: Tom H
authors: TH
authors: Greene
authors: Milena K
authors: MK
authors: Radeva
authors: Jennifer J
authors: JJ
authors: Gassman
authors: Marva M
authors: MM
authors: Moxey-Mims
authors: Ronald J
authors: RJ
authors: Hogg
authors: Sandra L
authors: SL
authors: Watkins
authors: Richard N
authors: RN
authors: Fine
authors: Susan L
authors: SL
authors: Hogan
authors: John P
authors: JP
authors: Middleton
authors: V Matti
authors: VM
authors: Vehaskari
authors: Patti A
authors: PA
authors: Flynn
authors: Leslie M
authors: LM
authors: Powell
authors: Suzanne M
authors: SM
authors: Vento
authors: June L
authors: JL
authors: McMahan
authors: Norman
authors: N
authors: Siegel
authors: Vivette D
authors: VD
authors: D'Agati
authors: Aaron L
authors: AL
authors: Friedman
mesh_terms: D000293:Adolescent; D000328:Adult; D001794:Blood Pressure / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000009:adverse effects / Q000627:therapeutic use; D003907:Dexamethasone / Q000008:administration & dosage / Q000009:adverse effects; D004359:Drug Therapy, Combination; D005260:Female; D005919:Glomerular Filtration Rate; D005923:Glomerulosclerosis, Focal Segmental / Q000188:drug therapy* / Q000503:physiopathology; D006801:Humans; D008297:Male; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives; D011446:Prospective Studies; D055815:Young Adult
chemical_list: D003907:Dexamethasone; D016572:Cyclosporine; D009173:Mycophenolic Acid