Key result
Steroid therapy in Duchenne muscular dystrophy is linked to ~76% lower mortality versus no steroids.
Why the study?
Although steroids slow musculoskeletal impairment in Duchenne muscular dystrophy, their effects on cardiac function and mortality remain unknown.
Does steroid therapy reduce mortality and new-onset cardiomyopathy in patients with Duchenne muscular dystrophy treated with RAAS antagonists?
Comparison
Steroid therapy vs no steroid therapy
Design
Cohort study
Follow-up
11.3 ± 4.1 years
Authors
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Hypothesis-generating for steroids in Duchenne muscular dystrophy; randomized trials needed before practice change.
Cohort (n=86)
Does steroid therapy reduce mortality and new-onset cardiomyopathy in patients with Duchenne muscular dystrophy treated with RAAS antagonists?
Hazard Ratio: 0.24 (95% CI 0.07–0.91)
Absolute Event Rate: 11% vs 43%
p-value: p=0.0351
In patients with Duchenne muscular dystrophy, prophylactic steroid therapy is associated with significantly reduced all-cause mortality and a lower incidence of new-onset and progressive cardiomyopathy.
Schram et al. (2013) conducted a cohort in Duchenne muscular dystrophy (n=86). Steroid therapy vs. No steroid therapy was evaluated on Mortality rate (HR 0.24, 95% CI 0.07 to 0.91, p=0.0351). Steroid therapy in patients with Duchenne muscular dystrophy was associated with a 76% lower mortality rate (HR 0.24; 95% CI 0.07-0.91; p=0.0351) compared to no steroid therapy.
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