心肌肌球蛋白抑制剂治疗肥厚型心肌病的研究进展

袁叶; 杨珍珍; 于博; 孙倩; 张志清

doi:10.6039/j.issn.1001-0408.2023.21.20

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心肌肌球蛋白抑制剂治疗肥厚型心肌病的研究进展

综述 | 更新时间：2023-11-10

- 心肌肌球蛋白抑制剂治疗肥厚型心肌病的研究进展
- Advances in the treatment of hypertrophic cardiomyopathy with cardiac myosin inhibitors
- 中国药房 2023年34卷第21期页码：2684-2688
- 作者机构：
  
  河北医科大学第二医院药学部，石家庄　050000
- 作者简介：
  
  主管药师，硕士。研究方向：心血管内科临床药学及体内药物分析。E-mail：243185935@qq.com
  主任药师，硕士生导师，博士。研究方向：体内药物分析及相互作用、临床药学、群体药动学及个体化给药。E-mail：777yyy@sina.cn
- 基金信息：
  
  河北省医学科学研究课题(20200062)
- DOI：10.6039/j.issn.1001-0408.2023.21.20
  中图分类号： RR972;R96
- 纸质出版日期：2023-11-15，
  
  收稿日期：2023-04-07，
  
  修回日期：2023-09-25，
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袁叶,杨珍珍,于博等.心肌肌球蛋白抑制剂治疗肥厚型心肌病的研究进展 ^Δ[J].中国药房,2023,34(21):2684-2688.

YUAN Ye,YANG Zhenzhen,YU Bo,et al.Advances in the treatment of hypertrophic cardiomyopathy with cardiac myosin inhibitors[J].ZHONGGUO YAOFANG,2023,34(21):2684-2688.
袁叶,杨珍珍,于博等.心肌肌球蛋白抑制剂治疗肥厚型心肌病的研究进展 ^Δ[J].中国药房,2023,34(21):2684-2688. DOI： 10.6039/j.issn.1001-0408.2023.21.20.

YUAN Ye,YANG Zhenzhen,YU Bo,et al.Advances in the treatment of hypertrophic cardiomyopathy with cardiac myosin inhibitors[J].ZHONGGUO YAOFANG,2023,34(21):2684-2688. DOI： 10.6039/j.issn.1001-0408.2023.21.20.

摘要

随着学界对肥厚型心肌病遗传机制的不断了解，最新上市的针对肥厚型心肌病基因突变的新型分子靶向药物Mavacamten和Aficamten均被美国FDA批准用于治疗肥厚型梗阻性心肌病。Mavacamten和Aficamten的作用机制相似，均可选择性地与心肌肌球蛋白的不同变构位点结合来抑制心肌肌球蛋白，从而减轻心肌的高收缩性。相关临床研究表明，2种药物均可降低患者左心室流出道压力梯度、降低心脏标志物N末端B型利钠肽原和心肌肌钙蛋白I水平，并在一定程度上改善纽约心脏协会心功能分级，且安全性良好、不良反应轻、患者均可耐受。相比于Mavacamten，Aficamten作为结构优化后的产物，具有较短的半衰期及更少的药物相互作用，更利于药物目标剂量滴定。

Abstract

In recent years， with the increasing understanding of the genetic mechanisms of hypertrophic cardiomyopathy， novel molecular-targeted drugs Mavacamten and Aficamten are two cardiac myosin inhibitors currently approved by the FDA for the treatment of hypertrophic obstructive cardiomyopathy. Both of them have a similar mechanism of action and can selectively bind to different variable sites of cardiac myosin to inhibit cardiac myosin， thus reducing myocardial hypercontractility. Relevant clinical studies have also shown that both drugs can reduce patients’ left ventricular outflow tract pressure gradient， the levels of N-terminal pro-B-type natriuretic peptide and cardiac troponin I as cardiac markers， and improve New York Heart Association （NYHA） cardiac function class. They are safe， have mild adverse reactions， and can be tolerated by patients. Compared to Mavacamten， Aficamten， as a structurally optimized product， has a shorter half-life and fewer drug-drug interactions， which is more conducive to drug-targeted dose titration.

关键词

心肌肌球蛋白抑制剂肥厚型梗阻性心肌病MavacamtenAficamten分子靶向药物

Keywords

hypertrophic obstructive cardiomyopathyMavacamtenAficamtenmolecular-targeted drugs

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