McDonald CM, Sajeev G, Yao Z, et al. Deflazacort versus prednisone treatment for Duchenne muscular dystrophy: a meta-analysis of disease progression rates in recent multicenter clinical trials. Muscle & nerve 2019.
BINTRODUCTION: We characterized disease progression over 48 weeks among boys receiving deflazacort versus 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 >/= 6 months of prior corticosteroid use and stable baseline dosing. Associations between corticosteroid 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 versus prednisone/prednisolone-treated patients experienced, on average, lower declines of 28.3 meters on six-minute walk distance [95% confidence interval: (5.7, 50.9)], 2.9 seconds on rise from supine, [(0.9, 4.9)], 2.3 seconds on 4 stair climb [(0.5, 4.1)], and 2.9 points [(0. 1, 5.8)] on the North Star Ambulatory Assessment linearized score.
DISCUSSION: Deflazacort-treated patients experienced significantly lower functional decline over 48 weeks.