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

Shieh PB, McIntosh J, Jin F, et al. Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial. Muscle & nerve 2018;58:639-645

Shieh_2018_Muscle Nerve. Nov;58(5);639-645

This study compared retrospectively the effects on physical function of the corticosteroids prednisone/prednisolone and deflazacort, by looking at patients receiving placebo from the ataluren (stop codon readthrough) study. Results seem in favour of deflazacort were less decline of muscle function (walking distance, muscle function tests) and less increase in weight and BMI, but their growth (increase in height) was also lower. The results should, however, be interpreted with caution since the analysis was done retrospectively and the original study did not aim to look at the effect of corticosteroids.

 

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.

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