Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials.

ABSTRACT: INTRODUCTION:In this study we characterized disease progression over 48?weeks among boys receiving deflazacort vs prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials. METHODS:Ambulatory boys with DMD receiving placebo in the phase 3 ataluren (N = 115) and tadalafil (N = 116) trials were included. The trials required at least 6?months of prior corticosteroid use and stable baseline dosing. Associations between corticosteroid use and 48-week changes in ambulatory function were estimated using mixed models. Adjusted differences between corticosteroid groups were pooled in a meta-analysis. RESULTS:In the meta-analysis, deflazacort-treated patients vs prednisone/prednisolone-treated patients experienced, on average, lower declines of 28.3 meters on 6-minute walk distance (95% confidence interval [CI], 5.7, 50.9; 2.9?seconds on rise from supine [95% CI, 0.9, 4.9?seconds]; 2.3?seconds on 4-stair climb [95% CI, 0.5, 4.1?seconds]; and 2.9 [95% CI, 0.1, 5.8] points on the North Star Ambulatory Assessment linearized score). DISCUSSION:Deflazacort-treated patients experienced significantly lower functional decline over 48?weeks.

SUBMITTER: McDonald CM 

PROVIDER: S-EPMC6973289 | biostudies-literature | 2020 Jan

REPOSITORIES: biostudies-literature