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Randomized Controlled Trial
. 2018 Nov;58(5):639-645.
doi: 10.1002/mus.26191. Epub 2018 Sep 27.

Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial

Perry B Shieh  1 , Joseph Mcintosh  2 , Fengbin Jin  2 , Marcio Souza  2 , Gary Elfring  2 , Siva Narayanan  2 , Panayiota Trifillis  2 , Stuart W Peltz  2 , Craig M Mcdonald  3 , Basil T Darras  4 ; THE ACT DMD STUDY GROUP
Affiliations
Randomized Controlled Trial

Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial

Perry B Shieh et al. Muscle Nerve. 2018 Nov.

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-minute walk 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 comparison will better define these differences. Muscle Nerve 58: 639-645, 2018.

Keywords: deflazacort; muscular dystrophy; prednisolone; prednisone; walking.

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Figures

Figure 1
Figure 1
Least‐square mean changes and t‐statistics from baseline to week 48 in assessments of physical functioning and HRQoL (ITT) for deflazacort versus prednisone/prednisolone.
See this image and copyright information in PMC

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