title: Clinical trials treating focal segmental glomerulosclerosis should measure patient quality of life.
abstract: Optimal therapy of patients with steroid-resistant primary focal segmental glomerulosclerosis (FSGS) remains controversial. This report describes the initial study design, baseline characteristics, and quality of life of patients enrolled in the FSGS Clinical Trial, a large multicenter randomized study of this glomerulopathy comparing a 12-month regimen of cyclosporine to the combination of mycophenolate mofetil and oral dexamethasone. Patients with age ranging 2-40 years, with an estimated glomerular filtration rate > 40 ml/min per 1.73 m², a first morning urine protein-to-creatinine ratio over one, and resistant to corticosteroids were eligible. The primary outcome was complete or partial remission of proteinuria over 52 weeks after randomization. In all, 192 patients were screened, of whom 138 were randomized for treatment. Ethnic distributions were 53 black, 78 white, and 7 other. By self- or parent-proxy reporting, 26 of the 138 patients were identified as Hispanic. The baseline glomerular filtration rate was 112.4 (76.5, 180.0) ml/min per 1.73 m², and urine protein was 4.0 (2.1, 5.3) g/g. Overall, the quality of life of the patients with FSGS was lower than healthy controls and similar to that of patients with end-stage renal disease. Thus, the impact of FSGS on quality of life is significant and this measurement should be included in all trials.
authors.affiliation: University of Michigan, Pediatrics & Communicable Diseases, Pediatric Nephrology, Ann Arbor, Michigan, USA. Electronic address: dgipson@umich.edu.
authors.forename: Debbie S
authors.initials: DS
authors.lastname: Gipson
authors.affiliation: Division of Nephrology, Department of Pediatrics, Cohen Children's Hospital, New York, New York, USA.
authors.forename: Howard
authors.initials: H
authors.lastname: Trachtman
authors.affiliation: Montefiore Medical Center, Children's Hospital, Pediatric Nephrology, New York, New York, USA.
authors.forename: Frederick J
authors.initials: FJ
authors.lastname: Kaskel
authors.affiliation: Cleveland Clinic, Department of Quantitative Health Sciences, Cleveland, Ohio, USA.
authors.forename: Milena K
authors.initials: MK
authors.lastname: Radeva
authors.affiliation: Cleveland Clinic, Department of Quantitative Health Sciences, Cleveland, Ohio, USA.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Gassman
authors.affiliation: Division of Epidemiology, University of Utah, Salt Lake City, Utah, USA.
authors.forename: Tom H
authors.initials: TH
authors.lastname: Greene
authors.affiliation: NIH/NIDDK, Division of Kidney, Urology & Hematology, Bethesda, Maryland, USA.
authors.forename: Marva M
authors.initials: MM
authors.lastname: Moxey-Mims
authors.affiliation: The Children's Hospital at Scott & White Medical Center, Department of Pediatrics, Temple, Texas, USA.
authors.forename: Ronald J
authors.initials: RJ
authors.lastname: Hogg
authors.affiliation: Division of Nephrology, Department of Pediatrics, University of Washington, Seattle, Washington, USA.
authors.forename: Sandra L
authors.initials: SL
authors.lastname: Watkins
authors.affiliation: Stony Brook University Medical Center, School of Medicine, Stony Brook, New York, USA.
authors.forename: Richard N
authors.initials: RN
authors.lastname: Fine
authors.affiliation: Division of Nephrology, Department of Medicine, Duke University, Durham, North Carolina, USA.
authors.forename: John P
authors.initials: JP
authors.lastname: Middleton
authors.affiliation: Louisiana State University Health Sciences Center and the Research Institute for Children, Children's Hospital & LSU Health Sciences Center, New Orleans, Louisiana, USA.
authors.forename: V M
authors.initials: VM
authors.lastname: Vehaskari
authors.affiliation: Division of Nephrology & Hypertension at Chapel Hill, UNC Kidney Center, University of North Carolina, Chapel Hill, North Carolina, USA.
authors.forename: Susan L
authors.initials: SL
authors.lastname: Hogan
authors.affiliation: Division of Nephrology, Department of Pediatrics, Cohen Children's Hospital, New York, New York, USA.
authors.forename: Suzzane
authors.initials: S
authors.lastname: Vento
authors.affiliation: Montefiore Medical Center, Children's Hospital, Pediatric Nephrology, New York, New York, USA.
authors.forename: Patti A
authors.initials: PA
authors.lastname: Flynn
authors.affiliation: Division of Nephrology & Hypertension at Chapel Hill, UNC Kidney Center, University of North Carolina, Chapel Hill, North Carolina, USA.
authors.forename: Leslie M
authors.initials: LM
authors.lastname: Powell
authors.affiliation: Cleveland Clinic, Department of Quantitative Health Sciences, Cleveland, Ohio, USA.
authors.forename: June L
authors.initials: JL
authors.lastname: McMahan
authors.forename: Norman
authors.initials: N
authors.lastname: Siegel
authors.affiliation: Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
authors.forename: Aaron L
authors.initials: AL
authors.lastname: Friedman
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000328:Adult; D016009:Chi-Square Distribution; D002648:Child; D002675:Child, Preschool; D003907:Dexamethasone / Q000008:administration & dosage*; D004351:Drug Resistance; D004359:Drug Therapy, Combination; D005260:Female; D005919:Glomerular Filtration Rate; D005923:Glomerulosclerosis, Focal Segmental / Q000150:complications / Q000188:drug therapy* / Q000208:ethnology / Q000523:psychology; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D008297:Male; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000031:analogs & derivatives*; D011446:Prospective Studies; D011507:Proteinuria / Q000188:drug therapy / Q000209:etiology; D020551:Pulse Therapy, Drug; D011788:Quality of Life; D012044:Regression Analysis; D012107:Research Design; D011795:Surveys and Questionnaires; D013997:Time Factors; D016896:Treatment Outcome; D014481:United States; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D003907:Dexamethasone; D009173:Mycophenolic Acid