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心肌肌球蛋白抑制剂在肥厚型心肌病管理中的临床应用:一项范围综述。

The clinical utility of cardiac myosin inhibitors for the management of hypertrophic cardiomyopathy: a scoping review.

作者信息

Othman Leen, Koskina Lida, Huerta Nicholas, Rao Shiavax J

机构信息

Department of Medicine, MedStar Union Memorial Hospital, Baltimore, MD, USA.

Division of Cardiovascular Medicine, University of Virginia, 1215 Lee St Box 800158, Charlottesville, VA, 22908, USA.

出版信息

Heart Fail Rev. 2025 Mar;30(2):453-467. doi: 10.1007/s10741-024-10476-w. Epub 2024 Dec 17.

DOI:10.1007/s10741-024-10476-w
PMID:39690360
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11802616/
Abstract

Hypertrophic cardiomyopathy (HCM) is an inherited condition characterized by left ventricular, non-dilated hypertrophy in the absence of another secondary underlying cause. There has been an ongoing increase in the diagnosis of HCM over the past couple of decades, prompting further work in the area of pharmacological and interventional therapies. This scoping review aimed to summarize the traditional therapeutic options for HCM and to explore emerging research on novel cardiac myosin inhibitors (CMIs) as a new option for pharmacologic management of HCM. A PRISMA search strategy was carried out to identify the pertinent literature on mavacamten and aficamten-two novel CMIs. Seventeen studies were included. Based on the results of the studies included in this review, cardiac myosin inhibitors have been proven to be a safe and efficacious second-line option for the management of HCM. In the foreseeable future, based on results of ongoing studies investigating patient outcomes and side-effect profile, CMIs may potentially play a larger role as part of standard treatment of HCM.

摘要

肥厚型心肌病(HCM)是一种遗传性疾病,其特征为左心室非扩张性肥厚,且不存在其他继发性潜在病因。在过去几十年中,HCM的诊断数量持续增加,这促使人们在药物和介入治疗领域开展进一步研究。本综述旨在总结HCM的传统治疗选择,并探索新型心肌肌球蛋白抑制剂(CMIs)作为HCM药物治疗新选择的新兴研究。采用PRISMA检索策略来识别有关mavacamten和aficamten这两种新型CMIs的相关文献。共纳入17项研究。基于本综述纳入研究的结果,心肌肌球蛋白抑制剂已被证明是治疗HCM的一种安全有效的二线选择。在可预见的未来,根据正在进行的研究中关于患者预后和副作用情况的结果,CMIs可能会在HCM的标准治疗中发挥更大作用。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b96e/11802616/7a72d4b80094/10741_2024_10476_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b96e/11802616/2e755631fe7b/10741_2024_10476_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b96e/11802616/7a72d4b80094/10741_2024_10476_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b96e/11802616/2e755631fe7b/10741_2024_10476_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b96e/11802616/7a72d4b80094/10741_2024_10476_Fig2_HTML.jpg

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本文引用的文献

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JAMA Cardiol. 2025 Feb 1;10(2):170-175. doi: 10.1001/jamacardio.2024.3810.
2
Mavacamten-Associated Temporal Changes in Left Atrial Function in Obstructive HCM: Insights From the VALOR-HCM Trial.梗阻性肥厚型心肌病中与马伐卡坦相关的左心房功能随时间的变化:来自VALOR-HCM试验的见解
JACC Cardiovasc Imaging. 2025 Mar;18(3):251-262. doi: 10.1016/j.jcmg.2024.08.005. Epub 2024 Sep 2.
3
Mavacamten in Obstructive Hypertrophic Cardiomyopathy Patients Referred for Septal Reduction: Health Status Analysis Through Week 56 in VALOR-HCM Trial.
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J Am Coll Cardiol. 2024 Sep 10;84(11):1041-1045. doi: 10.1016/j.jacc.2024.06.025.
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The Role of Multiple Mutations in Hypertrophic Cardiomyopathy - A New Universe to Discover: Proof of Guiltiness of the Genetic Burden in Worsening Hypertrophic Cardiomyopathy Natural History.多重突变在肥厚型心肌病中的作用——一个有待探索的全新领域:基因负荷在肥厚型心肌病自然病史恶化中“有罪”的证据
Cardiology. 2024;149(5):451-454. doi: 10.1159/000539360. Epub 2024 Jun 14.
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Sex-related differences in the association between septal wall thickness and survival.室间隔厚度与生存率之间关联的性别差异。
Int J Cardiol Heart Vasc. 2024 May 27;53:101427. doi: 10.1016/j.ijcha.2024.101427. eCollection 2024 Aug.
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