title: Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy.
abstract: OBJECTIVE
  To assess safety and efficacy of deflazacort (DFZ) and prednisone (PRED) vs placebo in Duchenne muscular dystrophy (DMD).
  
  
  METHODS
  This phase III, double-blind, randomized, placebo-controlled, multicenter study evaluated muscle strength among 196 boys aged 5-15 years with DMD during a 52-week period. In phase 1, participants were randomly assigned to receive treatment with DFZ 0.9 mg/kg/d, DFZ 1.2 mg/kg/d, PRED 0.75 mg/kg/d, or placebo for 12 weeks. In phase 2, placebo participants were randomly assigned to 1 of the 3 active treatment groups. Participants originally assigned to an active treatment continued that treatment for an additional 40 weeks. The primary efficacy endpoint was average change in muscle strength from baseline to week 12 compared with placebo. The study was completed in 1995.
  
  
  RESULTS
  All treatment groups (DFZ 0.9 mg/kg/d, DFZ 1.2 mg/kg/d, and PRED 0.75 mg/kg/d) demonstrated significant improvement in muscle strength compared with placebo at 12 weeks. Participants taking PRED had significantly more weight gain than placebo or both doses of DFZ at 12 weeks; at 52 weeks, participants taking PRED had significantly more weight gain than both DFZ doses. The most frequent adverse events in all 3 active treatment arms were Cushingoid appearance, erythema, hirsutism, increased weight, headache, and nasopharyngitis.
  
  
  CONCLUSIONS
  After 12 weeks of treatment, PRED and both doses of DFZ improved muscle strength compared with placebo. Deflazacort was associated with less weight gain than PRED.
  
  
  METHODS
  This study provides Class I evidence that for boys with DMD, daily use of either DFZ and PRED is effective in preserving muscle strength over a 12-week period.
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL. Robert_Griggs@URMC.Rochester.edu.
authors: Robert C
authors: RC
authors: Griggs
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: J Phillip
authors: JP
authors: Miller
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: Cheryl R
authors: CR
authors: Greenberg
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: Darcy L
authors: DL
authors: Fehlings
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: Alan
authors: A
authors: Pestronk
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: Jerry R
authors: JR
authors: Mendell
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: Richard T
authors: RT
authors: Moxley
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: Wendy
authors: W
authors: King
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: John T
authors: JT
authors: Kissel
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: Valerie
authors: V
authors: Cwik
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: Michel
authors: M
authors: Vanasse
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: Julaine M
authors: JM
authors: Florence
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: Shree
authors: S
authors: Pandya
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: Jordan S
authors: JS
authors: Dubow
authors: From the University of Rochester Medical Center (R.C.G., R.T.M., S.P.), NY; Washington University in St Louis (J.P.M., A.P., J.M.F.), MO; University of Manitoba and Children's Hospital Research Institute of Manitoba (C.R.G.), Winnipeg; Department of Paediatrics (D.L.F.), Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Canada; Nationwide Children's Hospital (J.R.M.); Ohio State University Wexner Medical Center (W.K., J.T.K.), Columbus; Muscular Dystrophy Association (V.C.), Marlton, NJ; CHU Sainte Justine (M.V.), Montreal, Canada; and Marathon Pharmaceuticals, LLC (J.S.D., J.M.M.), Northbrook, IL.
authors: James M
authors: JM
authors: Meyer
mesh_terms: D000293:Adolescent; D000893:Anti-Inflammatory Agents / Q000009:adverse effects / Q000627:therapeutic use*; D001835:Body Weight / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D006801:Humans; D016018:Least-Squares Analysis; D008297:Male; D009043:Motor Activity / Q000187:drug effects; D053580:Muscle Strength / Q000187:drug effects; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology; D011241:Prednisone / Q000009:adverse effects / Q000627:therapeutic use*; D011282:Pregnenediones / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D000893:Anti-Inflammatory Agents; D011282:Pregnenediones; C021988:deflazacort; D011241:Prednisone