title: Utility of cystatin C to monitor renal function in Duchenne muscular dystrophy.
abstract: Creatinine as a marker of renal function has limited value in Duchenne muscular dystrophy (DMD) because of reduced muscle mass. Alternative methods of assessing renal function are sorely needed. Cystatin C, a nonglycosylated protein unaffected by muscle mass, is potentially an ideal biomarker of nephrotoxicity for this population but requires validation. In all, 75 subjects were recruited: 35 DMD (mean age 10.8 +/- 5.4 years, corticosteroids n = 19, ambulatory n = 26), 29 healthy controls, 10 with renal disease, and one DMD with renal failure. Cystatin C levels in DMD were normal irrespective of age, ambulation, or corticosteroid treatment. Serum cystatin C was 0.67 +/- 0.11 mg/l compared to normal controls 0.69 +/- 0.09. mg/l. In these same individuals serum creatinine was severely reduced (0.27 +/- 0.12 mg/dl) versus normals (0.75 +/- 0.15 mg/dl, P < 0.01). In one DMD subject in renal failure, cystatin C was elevated. This study demonstrates the potential value of cystatin C as a biomarker for monitoring renal function in DMD. Its applicability extends to other neuromuscular diseases.
authors.affiliation: Research Institute at Nationwide Children's Hospital and Department of Pediatrics at Ohio State University, 700 Children's Drive, Room 3011, Columbus, Ohio 43205, USA.
authors.forename: Laurence
authors.initials: L
authors.lastname: Viollet
authors.forename: Susan
authors.initials: S
authors.lastname: Gailey
authors.forename: David J
authors.initials: DJ
authors.lastname: Thornton
authors.forename: Neil R
authors.initials: NR
authors.lastname: Friedman
authors.forename: Kevin M
authors.initials: KM
authors.lastname: Flanigan
authors.forename: John D
authors.initials: JD
authors.lastname: Mahan
authors.forename: Jerry R
authors.initials: JR
authors.lastname: Mendell
mesh_terms: D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000097:blood; D002648:Child; D003404:Creatinine / Q000097:blood; D055316:Cystatin C / Q000097:blood*; D051341:Dependent Ambulation; D005260:Female; D006801:Humans; D052177:Kidney Diseases, Cystic / Q000097:blood* / Q000209:etiology*; D008297:Male; D008991:Monitoring, Physiologic / Q000379:methods; D020388:Muscular Dystrophy, Duchenne / Q000097:blood / Q000150:complications*
chemical_list: D000305:Adrenal Cortex Hormones; D055316:Cystatin C; D003404:Creatinine