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ACE Inhibitors in Duchenne Muscular Dystrophy
Quick Takes
- There is a significant overall survival benefit and lower risk of hospitalization for the management of HF associated with prophylactic ACEi treatment in Duchenne muscular dystrophy patients with preserved LV function.
- This paper adds to previous literature that suggests mineralocorticoid receptor antagonists are beneficial in Duchenne muscular dystrophy patients.
Study Questions:
What is the effect of prophylactic angiotensin-converting enzyme inhibitors (ACEi) on survival in Duchenne muscular dystrophy (DMD)?
Methods:
The study authors analyzed the data from the French multicenter DMD Heart Registry. The authors estimated the association between the prophylactic prescription of ACEi and event-free survival in 668 DMD patients ages 8-13 years, with normal left ventricular (LV) function, using: 1) a Cox model with intervention as a time-dependent covariate, 2) a propensity-based analysis comparing ACEi treatment versus no treatment, and 3) a set of sensitivity analyses. The study outcomes were overall survival and hospitalizations for heart failure (HF) or acute respiratory failure.
Results:
Among 668 patients, 576 DMD patients (mean age 6.1 ± 2.8 years) were eligible for this study, of whom 390 were treated with ACEi prophylactically. Death occurred in 13.5% of the patients (n = 53) who were treated with ACEi and 32.3% of the patients (n = 60) who were not treated prophylactically with ACEi. In a Cox model with intervention as a time-dependent variable, the hazard ratio (HR) associated with ACEi treatment was 0.49 (95% confidence interval [CI], 0.34–0.72) and 0.47 (95% CI, 0.31–0.17) for overall mortality after adjustment for baseline variables. In the propensity-based analysis, 278 patients were included in the treatment group and 834 in the control group, with 18.5% and 30.4% 12-year estimated probability of death, respectively. ACEi were associated with a lower risk of death (HR, 0.39; 95% CI, 0.17–0.92) and hospitalization for HF (HR, 0.16; 95% CI, 0.04–0.62). All other sensitivity analyses yielded similar results.
Conclusions:
The study authors concluded that prophylactic ACEi therapy in DMD was associated with a significantly higher overall survival and lower rates of hospitalization for HF.
Perspective:
As the authors discuss in their paper, natural history studies in DMD patients have reported that end-stage HF developed in up to 40% of the patients in the second or third decades of life and, therefore, the findings of this study are important. These findings suggest that starting ACEi therapy should be an important quality metric in the management of DMD patients. Animal studies have suggested that mineralocorticoid receptor antagonists may have a similar effect (Circulation 2011;124:582-8; Orphanet J Rare Dis 2017;12:39).
Clinical Topics: Cardiovascular Care Team, Heart Failure and Cardiomyopathies, Prevention, Acute Heart Failure
Keywords: Angiotensin-Converting Enzyme Inhibitors, Cardiomyopathies, Heart Failure, Mineralocorticoid Receptor Antagonists, Muscular Dystrophy, Duchenne, Pediatrics, Respiratory Insufficiency, Secondary Prevention, Survival, Ventricular Function, Left
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