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 m 2, 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 m 2, 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.
Bibliographical noteFunding Information:
This study was sponsored by the NIH/NIDDK grants U01—DK063385, DK063490, DK063455, and DK063549. A portion of the pediatric quality-of-life data were previously published in an abstract form by the American Society of Nephrology Renal Week, 2009. This study represents the participation of the following institutions and investigators: Anjali Acharia, Jacobi Medical Center; Steven Alexander, Stanford Children’s Hospital; Amira Al-Uzri, Doernbecher Children’s Hospital, Oregon; Sharon Andreoli, Indiana University Riley Hospital; Gerald Appel, Columbia University Presbyterian Medical Center –Internal Medicine; Mazen Arar, University of Texas Health Science Center at San Antonio; Bettina Ault, University of Tennessee Health Science, Memphis; William Baker, Blue Ridge Nephrology Associates; Noosha Baqi, Saint Barnabas Medical Center, NJ; Sharon Bartosh, University of Wisconsin; Lorraine Bell, Montreal Children’s Hospital; Nadine Benador, University of California San Diego; Corrine Benchimol, Mount Sinai School of Medicine–Peds; Mark Benfield, University of Alabama at Birmingham; Danilo Bernardo, Valley Hypertension Nephrology; Phillip Berry, Specially for Children; Andrew Bland, Renalcare Associates of Peoria, IL; Tom Blydt-Hansen, Children’s Hospital, Winnipeg; Karl Brandspigel, Albemarle Nephrology Associates; Milos Budisavljevic, Medical University of South Carolina; Brad Carter, Kidney Specialists of Central Oklahoma; Daniel Cattran, Toronto General Hospital; Deepa Chand, Cleveland Clinic; Chandran Chandra, St Joseph Regional Medical Center; Kaye Christopher, Nephrology Associates of Hartsville; Howard Corey, Morristown Memorial Hospital, NJ; Eileen Ellis, Arkansas Children’s Hospital; Demetrius Ellis, Children’s Hospital of Pittsburgh; Babatunde Fariyike, Babatunde Fariyike Nephrology; Robert Fildes, Fairfax Hospital for Children; Elie Firzli, St Joseph’s Hospital and Medical Center; Danny Fischer, Kidney and Hypertension Center, Cincinnati; Joseph Flynn, Children’s Hospital, Seattle; John Foreman, Duke University Medical Center–Peds; Susan Furth, Johns Hopkins Pediatrics Nephrology; Debbie Gipson, University of North Carolina, Chapel Hill; Larry Greenbaum, Emory University; Ted Groshong, University of Missouri–Columbia; Anne Guillot, University of Vermont; Robert A. Gutman, Durham Nephrology; German Hernandez, Texas Tech University Health Sciences Center; Jeffrey Hoggard, Eastern Nephrology Associates; Robert Holleman, University of South Carolina; Mohammad Ilyas, University of Florida Jacksonville; J. Ashley Jefferson, University of Washington Medical Center; Eunice John, University of Illinois at Chicago; Valerie Johnson, Cornell Medical College New York Presbyterian Hospital; Joshua Kaplan, University of Medicine and Dentistry of New Jersey; Frederick Kaskel, Montefiore Medical Center; Michelle Krause, University of Arkansas, Internal Medicine Nephrology; Juan Kupferman, Maimonides Medical Center; Marc Lande, University of Rochester; Jerome Lane, Children’s Memorial Hospital, Chicago; Gary Lerner, Children’s Hospital Los Angeles; Jen-Jar Lin, University of Michigan; John Mahan, Ohio State University–Peds; Susan Massengill, Carolinas Medical Center–Peds; Douglas Matsell, Children’s Hospital British Columbia; Tej Mattoo, Children’s Hospital of Michigan; Lawrence Mcgee, Spartanburg Nephrology; Susan Mendley, University of Maryland Hospital; Richard Merrill, Kidney Center, Greenville, NC; John Middleton, Duke University Medical Center–Internal Medicine; Julian Midgley, Alberta Children’s Hospital; Asha Moudgil, Children’s National Medical Center; Dianne Muchant, University of Louisville; Jerome Murphy, Children’s Hospital Central California; James Musgrave, University of Hawaii School of Medicine; Martin Nash, Columbia University Babies’ And Children’s Hospital; Richard Neiberger, University of Florida, Gainesville; Victoria Norwood, University of Virginia Children’s Medical Center; Luis Ortiz, Medical College of Georgia; Cynthia Pan, Children’s Hospital of Wisconsin; Ana Paredes, Miami Children’s Hospital; Rupal Patel, Baylor College of Medicine–Internal Medicine; Dechu Puliyanda, Cedars-Sinai Medical Center; Majid Rasoulpour, Connecticut Children’s Medical Center; Shobha Sahney, Loma Linda University; Virginia Savin, Medical College of Wisconsin; Jon Scheinman, University of Kansas; Jeffrey Schelling, Case Western Reserve University, Internal Medicine, Nephrology; Morris Schoeneman, Suny Health Sci Center at Brooklyn; Mouin Seikaly, Children’s Medical Center of Dallas; Muhammed Shakeel, Nephrology and Internal Medicine, Anderson, SC; Ganesh Shidham, Ohio State University Medical Center; Ajay Singh, Brigham and Women’s Hospital; Michael Somers, Children’s Hospital Boston; James Springate, Women’s and Children’s Hospital of Buffalo; Frederic Strife, Cincinnati Children’s Hospital Medical Center; Rita Swinford, University of Texas Southwestern Houston–Internal Medicine; Howard Trachtman, Cohen Children’s Medical Center, NY; Martin Turman, University of Oklahoma Health Sciences Center; Matti Vehaskari, Children’s Hospital, New Orleans; Brad Warady, Children’s Mercy Hospital, Kansas City, MO; Steven Wassner, Penn State Children’s Hospital; Lynne Weiss, Robert Wood Johnson Medical School, NJ; Robert Weiss, Westchester Medical Center, NY; Dilys Whyte, SUNY at Stony Brook; Craig Wong, UNM Children’s Hospital; Ellen Wood, Cardinal Glennon Children’s Hospital in St Louis; Jonathon Woods, Southeastern Neph Associates Wilmington; Ora Yadin, Mattel’s Children’s Hospital UCLA; Verna Yiu, University of Alberta Pediatric Nephrology, Edmonton; Gaston Zilleruelo, University of Miami.
- focal segmental glomerulosclerosis
- quality of life
- randomized controlled trial