title: Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy.
abstract: OBJECTIVE
  To perform a double-blind, randomized study comparing efficacy and safety of daily and weekend prednisone in boys with Duchenne muscular dystrophy (DMD).
  
  
  METHODS
  A total of 64 boys with DMD who were between 4 and 10 years of age were randomized at 1 of 12 centers of the Cooperative International Neuromuscular Research Group. Efficacy and safety of 2 prednisone schedules (daily 0.75 mg/kg/day and weekend 10 mg/kg/wk) were evaluated over 12 months.
  
  
  RESULTS
  Equivalence was met for weekend and daily dosing of prednisone for the primary outcomes of quantitative muscle testing (QMT) arm score and QMT leg score. Secondary strength scores for QMT elbow flexors also showed equivalence between the 2 treatment groups. Overall side effect profiles of height and weight, bone density, cataract formation, blood pressure, and behavior, analyzed at 12 months, did not differ between weekend and daily dosing of prednisone.
  
  
  CONCLUSIONS
  Weekend dosing of prednisone is equally beneficial to the standard daily dosing of prednisone. Analysis of side effect profiles demonstrated overall tolerability of both dosing regimens.
  
  
  METHODS
  This study provides Class I evidence that weekend prednisone dosing is as safe and effective as daily prednisone in preserving muscle strength and preventing body mass index increases in boys with DMD over a 12-month period.
authors.affiliation: Children's National Medical Center, Washington, DC, USA. diana.escolar@gmail.com
authors.forename: D M
authors.initials: DM
authors.lastname: Escolar
authors.forename: L P
authors.initials: LP
authors.lastname: Hache
authors.forename: P R
authors.initials: PR
authors.lastname: Clemens
authors.forename: A
authors.initials: A
authors.lastname: Cnaan
authors.forename: C M
authors.initials: CM
authors.lastname: McDonald
authors.forename: V
authors.initials: V
authors.lastname: Viswanathan
authors.forename: A J
authors.initials: AJ
authors.lastname: Kornberg
authors.forename: T E
authors.initials: TE
authors.lastname: Bertorini
authors.forename: Y
authors.initials: Y
authors.lastname: Nevo
authors.forename: T
authors.initials: T
authors.lastname: Lotze
authors.forename: A
authors.initials: A
authors.lastname: Pestronk
authors.forename: M M
authors.initials: MM
authors.lastname: Ryan
authors.forename: E
authors.initials: E
authors.lastname: Monasterio
authors.forename: J W
authors.initials: JW
authors.lastname: Day
authors.forename: A
authors.initials: A
authors.lastname: Zimmerman
authors.forename: A
authors.initials: A
authors.lastname: Arrieta
authors.forename: E
authors.initials: E
authors.lastname: Henricson
authors.forename: J
authors.initials: J
authors.lastname: Mayhew
authors.forename: J
authors.initials: J
authors.lastname: Florence
authors.forename: F
authors.initials: F
authors.lastname: Hu
authors.forename: A M
authors.initials: AM
authors.lastname: Connolly
mesh_terms: D000367:Age Factors; D015992:Body Mass Index; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D004334:Drug Administration Schedule; D005500:Follow-Up Studies; D005938:Glucocorticoids / Q000008:administration & dosage*; D006801:Humans; D008297:Male; D053580:Muscle Strength / Q000187:drug effects; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology; D011241:Prednisone / Q000008:administration & dosage*; D016896:Treatment Outcome
chemical_list: D005938:Glucocorticoids; D011241:Prednisone