Comparative efficacy of different pharmacotherapies for muscular dystrophy: a systematic review and network meta-analysis
Abstract
Background: Muscular dystrophies (MDs) are rare neuromuscular disorders that lead to progressive muscle degeneration and early death. MDs like Duchenne have a poor prognosis associated with an unclear preferred treatment. Despite several new agents being assessed in clinical trials, no network meta-analysis (NMA) has been conducted to date evaluating different treatment options for MDs. Methods: MEDLINE, EMBASE, and Cochrane databases were searched for randomized and non-randomized clinical trials testing different agents in patients with any type of MD. The NMA was performed under a Frequentist framework using RStudio software version 4.3.2 and extension packages “netmeta” and “dmetar” for all statistical analysis. Our primary outcome was the improvement in 6-minute walk distance (6MWD) and the change in time to run/walk 10 m (TTRW). The NMA pooled all the direct and indirect evidence and simultaneously compared several treatments using the network relationship through a model for continuous outcomes. Results: 17 studies and 1,905 patients were included, of whom 1,802 (94.59%) were diagnosed with Duchenne muscular dystrophy. Among the included studies, 11 different drugs were evaluated for their efficacy in improving disease progression. Ataluren and drisapersen significantly increased the 6-minute walk distance in comparison with placebo: (SMD -21.08; 95% CI -23.08 to -19.08) and (SMD -12.91; 95% CI -15.32 to -10.51), respectively. Additionally, no benefit was shown with…
How strong is the evidence?
Evidence Hub reads this as a meta-analysis, done in people. Its evidence level is 1 of 5 (Highest · pooled evidence). Pooled evidence from several studies sits at the top of the evidence pyramid. Reported size: 1,905 participants or records.
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