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

Muscle Nerve. 2020 Jan;61(1):26-35. doi: 10.1002/mus.26736. Epub 2019 Nov 7.

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.

Keywords: Duchenne muscular dystrophy; ambulatory function; deflazacort; meta-analysis; prednisone/prednisolone.

Publication types

  • Meta-Analysis
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anti-Inflammatory Agents / therapeutic use*
  • Child
  • Disease Progression
  • Humans
  • Male
  • Multicenter Studies as Topic
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Prednisolone / therapeutic use
  • Prednisone / therapeutic use*
  • Pregnenediones / therapeutic use*
  • Randomized Controlled Trials as Topic
  • Treatment Outcome
  • Walking

Substances

  • Anti-Inflammatory Agents
  • Pregnenediones
  • Prednisolone
  • deflazacort
  • Prednisone