Department of Cardiology, University of Campania "L. Vanvitelli", Monaldi Hospital, Naples, Italy.
Medical Genetics and Cardiomyology, University of Campania "L. Vanvitelli", Naples, Italy.
Acta Myol. 2020 Sep 1;39(3):136-140. doi: 10.36185/2532-1900-018. eCollection 2020 Sep.
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.
营养不良型心肌病（DCM）是贝克型肌营养不良症（BMD）患者的常见表现，显著增加了发病率和死亡率。心肌几乎完全被纤维和脂肪结缔组织取代，导致不可逆的心力衰竭，其特征是射血分数逐渐降低。根据 PARADIGM-HF 试验结果，欧洲心脏病学会（ESC）指南建议在射血分数降低且仍有症状的心力衰竭门诊患者中使用沙库巴曲缬沙坦。迄今为止，关于沙库巴曲缬沙坦在 DCM 中的应用知之甚少。我们报告了一例肌营养不良型终末期扩张型心肌病、射血分数降低的患者，他对沙库巴曲缬沙坦治疗有反应。
