Hypertrophic Cardiomyopathy: From Medical Treatment to Advanced Heart Failure Therapies.

IF 4.3 3区材料科学 Q1 ENGINEERING, ELECTRICAL & ELECTRONIC

ACS Applied Electronic Materials Pub Date : 2024-09-01 Epub Date: 2024-07-11 DOI:10.1007/s11886-024-02095-6

Matylda Mazur, Wojciech Braksator, Eric Popjes

{"title":"Hypertrophic Cardiomyopathy: From Medical Treatment to Advanced Heart Failure Therapies.","authors":"Matylda Mazur, Wojciech Braksator, Eric Popjes","doi":"10.1007/s11886-024-02095-6","DOIUrl":null,"url":null,"abstract":"Purpose of review: There has been much debate surrounding novel medical therapies and heart transplantation listing challenges in patients with hypertrophic cardiomyopathy (HCM).Recent findings: Recent clinical trials led to FDA approval of mavacamten (a cardiac myosin inhibitor), offering symptom relief and potentially delaying/avoiding invasive septal reduction therapies for some patients with HCM and left ventricular outflow obstruction (LVOTO). For those with refractory symptoms and end-stage heart failure, heart transplantation remains the gold standard. However, the concern for the organ allocation system failing to prioritize those individuals persists. HCM is a heterogeneous genetic condition with variable penetration and clinical presentation. Even though a large portion of patients remain asymptomatic, an important minority develops debilitating symptoms refractory to medical therapy. Post-HT short- and long-term outcomes are favorable. However, HT waitlist mortality remains high. For highly selected patients with HCM, a left ventricular assist device is a viable option.","PeriodicalId":3,"journal":{"name":"ACS Applied Electronic Materials","volume":null,"pages":null},"PeriodicalIF":4.3000,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"ACS Applied Electronic Materials","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s11886-024-02095-6","RegionNum":3,"RegionCategory":"材料科学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/7/11 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"ENGINEERING, ELECTRICAL & ELECTRONIC","Score":null,"Total":0}

引用次数: 0

Abstract

Purpose of review: There has been much debate surrounding novel medical therapies and heart transplantation listing challenges in patients with hypertrophic cardiomyopathy (HCM).

Recent findings: Recent clinical trials led to FDA approval of mavacamten (a cardiac myosin inhibitor), offering symptom relief and potentially delaying/avoiding invasive septal reduction therapies for some patients with HCM and left ventricular outflow obstruction (LVOTO). For those with refractory symptoms and end-stage heart failure, heart transplantation remains the gold standard. However, the concern for the organ allocation system failing to prioritize those individuals persists. HCM is a heterogeneous genetic condition with variable penetration and clinical presentation. Even though a large portion of patients remain asymptomatic, an important minority develops debilitating symptoms refractory to medical therapy. Post-HT short- and long-term outcomes are favorable. However, HT waitlist mortality remains high. For highly selected patients with HCM, a left ventricular assist device is a viable option.

Abstract Image

查看原文本刊更多论文

肥厚型心肌病：从药物治疗到先进的心力衰竭疗法。

综述的目的：围绕肥厚型心肌病（HCM）患者的新型医疗疗法和心脏移植上市难题一直存在许多争论：最近的临床试验促使美国食品与药物管理局批准了马伐康坦（一种心脏肌球蛋白抑制剂），为一些肥厚型心肌病和左心室流出道梗阻（LVOTO）患者缓解了症状，并有可能推迟/避免了侵入性室间隔缩小疗法。对于有难治性症状和终末期心力衰竭的患者，心脏移植仍是金标准。然而，器官分配系统未能优先考虑这些患者的问题依然存在。HCM 是一种异质性遗传病，具有不同的渗透性和临床表现。尽管大部分患者仍无症状，但也有少数患者会出现药物治疗难治的衰弱症状。HT 后的短期和长期预后良好。然而，HT 候诊死亡率仍然很高。对于经过严格筛选的 HCM 患者，左心室辅助装置是一种可行的选择。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

ACS Applied Electronic Materials Multiple-

CiteScore

7.20

自引率

4.30%

发文量

567