DEFLAZACORT VERSUS PREDNISONE/PREDNISOLONE FOR MAINTAINING MOTOR FUNCTION AND DELAYING LOSS OF AMBULATION: A POST HOC ANALYSIS FROM THE ACT DMD TRIAL

THE ACT DMD STUDY GROUP

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32 Scopus citations

Abstract

Introduction: ACT DMD was a 48-week trial of ataluren for nonsense mutation Duchenne muscular dystrophy (nmDMD). Patients received corticosteroids for ≥6 months at entry and stable regimens throughout study. This post hoc analysis compares efficacy and safety for deflazacort and prednisone/prednisolone in the placebo arm. Methods: Patients received deflazacort (n = 53) or prednisone/prednisolone (n = 61). Endpoints included change from baseline in 6-minutewalk distance (6MWD), timed function tests, estimated age at loss of ambulation (extrapolated from 6MWD). Results: Mean changes in 6MWD were -39.0 m (deflazacort; 95%confidence limit [CL], -68.85, -9.17) and -70.6 m (prednisone/prednisolone; 95% CL, -97.16, -44.02).Mean changes in 4-stair climb were 3.79 s (deflazacort; 95% CL, 1.54, 6.03) and 6.67 s (prednisone/prednisolone; 95% CL, 4.69, 8.64). Conclusions: This analysis, limited by its post hoc nature, suggests greater preservation of 6MWD and 4-stair climb with deflazacort vs. prednisone/prednisolone. A head-to-head comparisonwill better define these differences.

Original languageEnglish (US)
Pages (from-to)639-645
Number of pages7
JournalMuscle and Nerve
Volume58
Issue number5
DOIs
StatePublished - Nov 2018

Keywords

  • Deflazacort
  • Muscular dystrophy
  • Prednisolone
  • Prednisone
  • Walking

ASJC Scopus subject areas

  • Physiology
  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)

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