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马伐卡坦,肥厚性梗阻性心肌病的一种新型变革性疗法:文献综述。

Mavacamten, a novel revolutionizing therapy in hypertrophic obstructive cardiomyopathy: A literature review.

作者信息

Aguiar Tiago, Martins Elisabete

机构信息

Faculty of Medicine, University of Porto, Porto, Portugal.

Faculty of Medicine, University of Porto, Porto, Portugal; Department of Cardiology, São João University Hospital Center, Porto, Portugal.

出版信息

Rev Port Cardiol. 2022 Aug;41(8):693-703. doi: 10.1016/j.repc.2021.09.013. Epub 2022 Jul 19.

Abstract

Hypertrophic cardiomyopathy (HCM) is one of the most common inherited cardiac diseases, defined as a left ventricular wall thickness of ≥15 mm, in the absence of other causes of abnormal ventricular loading. A major hallmark of this disease is the presence of left ventricular outflow tract obstruction, which develops in up to three quarters of patients, referred to as obstructive hypertrophic cardiomyopathy. Current treatment is offered to symptomatic patients, based on the presence of documented left ventricular obstruction, aimed at reducing symptoms and disease progression. This is achieved through pharmacological empirical therapy, surgery, alcohol ablation and/or pacing. Mavacamten is a first-in-class allosteric inhibitor of cardiac myosin that promises to provide clinicians with targeted therapy for these patients. The aim of this review is to provide a general overview of the modern approach to the diagnosis and management of HCM, as well as to integrate all the current knowledge on mavacamten, in anticipation of a future change in the treatment algorithm of patients with HCM.

摘要

肥厚型心肌病(HCM)是最常见的遗传性心脏病之一,定义为左心室壁厚度≥15毫米,且不存在其他导致心室负荷异常的原因。该病的一个主要特征是左心室流出道梗阻,多达四分之三的患者会出现这种情况,称为梗阻性肥厚型心肌病。目前针对有症状的患者进行治疗,依据是有记录的左心室梗阻情况,旨在减轻症状并延缓疾病进展。这通过药物经验性治疗、手术、酒精消融和/或起搏来实现。马伐卡坦是首个心脏肌球蛋白变构抑制剂,有望为这些患者提供靶向治疗。本综述的目的是对HCM诊断和管理的现代方法进行总体概述,并整合有关马伐卡坦的所有现有知识,以期未来HCM患者的治疗方案有所改变。

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