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Neurology. 2016 Nov 15;87(20):2123-2131. Epub 2016 Aug 26.

Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy.

Author information

1
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.
2
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.

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.

CLASSIFICATION OF EVIDENCE:

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.

PMID:
27566742
PMCID:
PMC5109941
DOI:
10.1212/WNL.0000000000003217
[Indexed for MEDLINE]
Free PMC Article

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