Effect of Deflazacort and Prednisone Versus Placebo on Pulmonary Function in Boys with Duchenne Muscular Dystrophy Who Have Lost Ambulation | DuchenneXchange

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Abstracts & Posters

Effect of Deflazacort and Prednisone Versus Placebo on Pulmonary Function in Boys with Duchenne Muscular Dystrophy Who Have Lost Ambulation

key information

source: American Academy of Neurology

year: 2016

authors: Jordan Dubow, Timothy Cunniff, Stephen P. Wanaski, James Meyer

summary/abstract:

Objective:
To prospectively assess the effects of deflazacort and prednisone on pulmonary function in boys with DMD who have lost ambulation

Background:
Duchenne Muscular Dystrophy (DMD) is the most common type of muscular dystrophy. As the disease progresses, weakness of respiratory muscles results into restrictive pulmonary disease, pulmonary complications and increased morbidity and mortality.

Methods:
This randomized, double-blind, placebo-controlled, active comparator, Phase 3 study evaluated the efficacy of two deflazacort doses (0.9 mg/kg/day and 1.2 mg/kg/day) compared to prednisone (0.75 mg/kg/day) and placebo for the treatment of boys with DMD. The first segment compared deflazacort and prednisone to placebo over 12 weeks. The second segment compared the two doses of deflazacort to prednisone from 12 to 52 weeks. We conducted a post-hoc subgroup analysis of pulmonary function (FVC, MVV) in patients who were non-ambulatory at baseline.

Results:
A total of 196 participants from 9 centers were randomized to the 4 treatment groups; 45 patients were non-ambulatory at baseline. At 12 weeks, both doses of deflazacort demonstrated improvement over placebo on FVC and MVV (p=0.065 for 0.9 mg/kg/day on FVC and p=0.02 for 1.2 mg/kg/day on MVV) while prednisone had larger numerical declines than placebo in both measures. Over 52 weeks of treatment deflazacort at 0.9 mg/kg/day had a 0.128 L improvement in FVC and an 11.1 L/min improvement in MVV compared to prednisone (NS) while deflazacort at 1.2 mg/kg/day demonstrated a 13.6 L/min improvement in MVV over prednisone (p=0.065). More patients on deflazacort showed improvement from baseline in pulmonary function compared to prednisone.

Conclusions:
This is the first prospective, randomized, blinded study to demonstrate the benefits of deflazacort and prednisone on pulmonary function in non-ambulatory boys with DMD. Although some measures appeared to favor deflazacort over prednisone, well-powered clinical studies are needed to better assess these differences.

organisation: Cynapsus Therapeutics; Marathon Pharmaceuticals

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