title: Corticosteroids in Duchenne muscular dystrophy: major variations in practice.
abstract: BACKGROUND
  In 2004, a Cochrane Review and AAN practice parameter concluded that prednisone 0.75 mg/kg/day is of short-term efficacy in Duchenne muscular dystrophy (DMD). Subsequent efforts to standardize care for DMD indicated wide variation in corticosteroid use.
  
  
  METHODS
  We surveyed physicians who follow patients with DMD, including: (1) clinics in the TREAT-NMD (Translational Research in Europe-Assessment and Treatment of Neuromuscular Diseases) network (predominantly Europe) and (2) U.S. MDA clinic directors. We also documented the co-administered corticosteroids in a trial of a putative treatment (ataluren) for DMD.
  
  
  RESULTS
  Of 105 Treat-NMD clinicians, corticosteroids were not used in 10 clinics, and 29 different regimens were used--the most frequent 0.75 mg/kg/day prednisone (61 centers); 10 days on/10 days off (36 centers); 0.9 mg/kg/day deflazacort (32 centers); and 5 mg/kg/day on weekends (10 centers). Similar diversity was identified in MDA clinics and in the ataluren trial.
  
  
  CONCLUSIONS
  Variability in corticosteroid use suggests uncertainty about risks/benefits of corticosteroid regimens for DMD.
authors.affiliation: University of Rochester, Departments of Neurology and Biostatistics, 265 Crittenden Boulevard, CU 420669, Rochester, New York, 14642, USA. robert_griggs@urmc.rochester.edu
authors.forename: Robert C
authors.initials: RC
authors.lastname: Griggs
authors.forename: Barbara E
authors.initials: BE
authors.lastname: Herr
authors.forename: Allen
authors.initials: A
authors.lastname: Reha
authors.forename: Gary
authors.initials: G
authors.lastname: Elfring
authors.forename: Leone
authors.initials: L
authors.lastname: Atkinson
authors.forename: Valerie
authors.initials: V
authors.lastname: Cwik
authors.forename: Elaine
authors.initials: E
authors.lastname: McColl
authors.forename: Rabi
authors.initials: R
authors.lastname: Tawil
authors.forename: Shree
authors.initials: S
authors.lastname: Pandya
authors.forename: Michael P
authors.initials: MP
authors.lastname: McDermott
authors.forename: Kate
authors.initials: K
authors.lastname: Bushby
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D017322:Clinical Trials, Phase II as Topic / Q000379:methods; D003625:Data Collection / Q000379:methods; D005060:Europe / Q000453:epidemiology; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000453:epidemiology*; D010820:Physicians / Q000592:standards*; D017410:Practice Guidelines as Topic / Q000592:standards; D016032:Randomized Controlled Trials as Topic / Q000379:methods; D014481:United States / Q000453:epidemiology
chemical_list: D000305:Adrenal Cortex Hormones