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,

SUBMITTER: McDonald CM 

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

REPOSITORIES: biostudies-literature