Key result
Sacubitril/valsartan improved functional class, left ventricular ejection fraction, and ventricular remodeling in a patient with Duchenne muscular dystrophy-associated cardiomyopathy.
Why the study?
There has been no research regarding the effectiveness and safety of sacubitril/valsartan in patients with Duchenne muscular dystrophy-associated cardiomyopathy.
Does sacubitril/valsartan improve functional class and ventricular remodeling in a patient with DMD-associated cardiomyopathy and HFrEF?
Case Report (n=1)
Does sacubitril/valsartan improve functional class and ventricular remodeling in a patient with DMD-associated cardiomyopathy and HFrEF?
Sacubitril/valsartan may be effective in improving functional class and promoting reverse ventricular remodeling in patients with DMD-associated cardiomyopathy and HFrEF.
Hypothesis-generating for sacubitril/valsartan in DMD cardiomyopathy; should not yet change practice.
In individuals with an ejection fraction of 40% or less, the use of sacubitril/valsartan significantly lowers mortality or hospitalization. There has been no research related to the effectiveness and safety of sacubitril/valsartan in patients with Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. We hereby report a case of DMD-associated cardiomyopathy and heart failure with reduced ejection fraction who had been on routine guideline-directed medical treatment with no change in clinical or echocardiographic markers. When guideline-directed medical therapy was unsuccessful, sacubitril/valsartan was started which resulted in a significant change in functional class and significant ventricular remodeling, including an improvement in left ventricular (LV) ejection fraction, reduction in LV diastolic diameter, and a reduction in mitral regurgitation.
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Jariwala et al. (2022) conducted a case report in Duchenne muscular dystrophy-associated cardiomyopathy and heart failure with reduced ejection fraction (n=1). Sacubitril/Valsartan vs. Guideline-directed medical therapy was evaluated on Functional class and ventricular remodeling (LV ejection fraction, LV diastolic diameter, mitral regurgitation). Sacubitril/valsartan improved functional class, left ventricular ejection fraction, and ventricular remodeling in a patient with Duchenne muscular dystrophy-associated cardiomyopathy.
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