ACT DMD trial:  A post hoc comparing deflazacort vs prednisone/prednisolone

The 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. Here, the authors describe the results of a post hoc analysis that compared the efficacy and safety of deflazacort and prednisone/prednisolone in the placebo arm. 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 (LoA; extrapolated from 6MWD). The mean changes in 6MWD were -39.0 m (deflazacort; 95% 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).

Although limited by its post hoc nature, this analysis suggests greater preservation of 6MWD and 4-stair climb with deflazacort vs. prednisone/prednisolone. A head-to-head comparison will better define these differences.

Shieh PB, McIntosh J, Jin F, et al; ACT DMD study group. Deflazacort vs prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post hoc analysis from the ACT DMD trial. Muscle Nerve. 2018 Jul 20. doi: 10.1002/mus.26191. [Epub ahead of print]