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肥厚型梗阻性心肌病药物治疗的研究进展及未来方向

Review of Mavacamten for Obstructive Hypertrophic Cardiomyopathy and Future Directions.

机构信息

Section of Cardiovascular Imaging, Heart, Vascular and Thoracic Institute, Cleveland Clinic, Cleveland, OH, USA.

出版信息

Drug Des Devel Ther. 2023 Apr 8;17:1097-1106. doi: 10.2147/DDDT.S368590. eCollection 2023.

Abstract

Hypertrophic cardiomyopathy (HCM) is a condition with abnormal hypertrophy of the left ventricle in the absence of common causes. The most common form involves the basal septum and can lead to obstruction of the left ventricular outflow tract. Patients can experience exertional symptoms such as chest pain, dyspnea and syncope. Traditional treatment has included beta blockers and nondihydropyridine calcium channel blockers with second-line therapy being disopyramide. Recently, mavacamten, a cardiac myosin inhibitor, has demonstrated improvement in quantitative measures of obstruction and symptom relief to such a degree that patients were able to defer invasive management of the disease. This review focuses on the pharmacology of mavacamten, its clinical trial data and guidance on how to incorporate this drug into clinical practice. Furthermore, it discusses emerging therapies currently being investigated for HCM.

摘要

肥厚型心肌病(HCM)是一种在无常见病因的情况下左心室异常肥厚的病症。最常见的形式涉及基底间隔,并可导致左心室流出道阻塞。患者可能会出现胸痛、呼吸困难和晕厥等运动症状。传统的治疗方法包括β受体阻滞剂和非二氢吡啶类钙通道阻滞剂,二线治疗药物为双异丙吡胺。最近,心肌肌球蛋白抑制剂 mavacamten 已证明可改善梗阻的定量指标,并在一定程度上缓解症状,使患者能够推迟对该疾病的侵入性治疗。本综述重点介绍了 mavacamten 的药理学、临床试验数据以及将该药物纳入临床实践的指导意见。此外,它还讨论了目前正在研究用于治疗 HCM 的新兴疗法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1815/10094472/c5625faa7bec/DDDT-17-1097-g0001.jpg

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