Key result
Sacubitril/valsartan elicits a successful response in a patient with end-stage dystrophinopathic HFrEF.
Why the study?
Little is known about the use of sacubitril/valsartan in dystrophinopathic cardiomyopathy.
Does sacubitril/valsartan improve outcomes in a patient with end-stage dystrophinopathic cardiomyopathy?
Case Report (n=1)
Does sacubitril/valsartan improve outcomes in a patient with end-stage dystrophinopathic cardiomyopathy?
Sacubitril/valsartan may be a beneficial treatment option for patients with end-stage dystrophinopathic cardiomyopathy.
Hypothesis-generating for sacubitril/valsartan in dystrophinopathic cardiomyopathy; prospective data needed before adoption.
Dystrophinopathic cardiomyopathy (DCM) is an almost constant manifestation in Becker muscular dystrophy (BMD) patients significantly contributing to morbidity and mortality. The nearly complete replacement of the myocardium by fibrous and fatty connective tissue results in an irreversible cardiac failure, characterized by progressive reduction of the ejection fraction. According to PARADIGM-HF trial results, the European Society of Cardiology (ESC) guidelines recommend the use of sacubitril/valsartan in ambulatory patients with heart failure and reduced ejection fraction, who remain symptomatic despite an optimal medical therapy. To date, little is still known about the use of sacubitril/valsartan in DCM. We report the case of a patient with dystrophinopathic end stage dilated cardiomyopathy with reduced ejection fraction who successfully responded to sacubitril/valsartan treatment.
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Papa et al. (2020) conducted a case report in dystrophinopathic end stage dilated cardiomyopathy with reduced ejection fraction (n=1). sacubitril/valsartan was evaluated. One-month sacubitril/valsartan treatment elicited a successful response in a patient with dystrophinopathic end-stage dilated cardiomyopathy with reduced ejection fraction.
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