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Abstracts & Posters

A Comparison of the Effects of Deflazacort and Prednisone Versus Placebo on Timed Functional Tests in Boys with Duchenne Muscular Dystrophy

key information

source: American Academy of Neurology

year: 2016

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


To describe the timed motor performance of standing from lying position, climbing 4 stairs, and running or walking 30 feet in patients taking deflazacort, prednisone, or placebo.

The measurement of timed functional tests (TFTs) are commonly used to evaluate children with Duchenne muscular dystrophy (DMD). As muscle weakness progresses, compensatory movements are used to perform the tasks, resulting in higher measured times. Corticosteroids have demonstrated clinical benefit in the treatment of DMD including performance in timed function tests.

In a randomized, double-blind, placebo-controlled, active comparator, Phase 3 study, the efficacy of two doses of deflazacort (0.9 mg/kg/day and 1.2 mg/kg/day) was 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 an analysis of timed functional tests to compare treatment and placebo groups

TFTs showed statistically significant differences between active treatments and placebo in times from supine to standing, climbing 4 stairs, and time to run/walk 30 feet at 12 weeks (p<0.002). At 12 weeks there was a trend towards significant improvement with deflazacort 0.9 mg/kg/day vs. prednisone in time to climb 4 stairs (p=0.066). From baseline to 52 weeks, both doses of deflazacort had numerically greater improvement in TFTs compared to prednisone. However, only time to climb 4 stairs reached significance with deflazacort 0.9 mg/kg/day (p<0.05) and time to run/walk 30 feet trended towards significance with deflazacort 1.2 mg/kg/day (p=0.07) vs. prednisone.

Both deflazacort and prednisone demonstrated significant improvement versus placebo in timed functional testing. Benefits were sustained over 52 weeks with both deflazacort and prednisone, but deflazacort appeared to have some benefits compared to prednisone in certain motor functions.

organization: Marathon Pharmaceuticals, USA

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