臨床症例報告ジャーナル

Sacubitril/Valsartan for Heart Failure in Patients with Becker Muscular Dystrophy and Dilated Cardiomyopathy: A Case Series

Abstract

Maria Vittoria Matassini, Michela Coccia, Michela Aringolo, Matilda Shkoza, Francesca Lupidi, Mauro Silvestrini, Maria Gabriella Ceravolo, Marco Marini and Gian Piero Perna

Background: Dilated cardiomyopathy with heart failure is a common cause of morbidity and mortality in patients with Becker muscular dystrophy. An early diagnosis, treatment and a close follow-up are crucial in improving quality of life and prognosis. Neuro-hormonal therapy may improve symptoms and cardiac dysfunction and reduce mortality risk. Little is known about the use of sacubitril/valsartan for heart failure in patients with Becker muscular dystrophy and dilated cardiomyopathy.
Methods: We present a case series of four patients with Becker muscular dystrophy cardiomyopathy followed in a dedicated cardiologic neuromuscular program and treated with sacubitril/valsartan because of severe left ventricular dysfunction with heart failure symptoms.
Results: In our experience sacubitril/valsartan was effective in improving symptoms and functional capacity, in reducing hospital admission for HF and, when early introduced, it promoted positive reverse heart remodeling. Despite a fragile population, sacubitril/valsartan was safe, without episodes of hypotension or renal function worsening.
Conclusion: Sacubitril/valsartan may be considered an effective and safe pharmacological option in patients with Becker muscular dystrophy and dilated cardiomyopathy with reduced ejection fraction.