Current Landscape of Therapies for Transthyretin Amyloid Cardiomyopathy

IF 10.3 1区医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS

JACC. Heart failure Pub Date : 2025-05-01 DOI:10.1016/j.jchf.2025.03.017

Jan M. Griffin MD , Justin L. Grodin MD, MPH , Frederick L. Ruberg MD , Ahmad Masri MD, MS , Mazen Hanna MD , Mathew S. Maurer MD

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Abstract

Transthyretin amyloid cardiomyopathy (ATTR-CM) is an infiltrative cardiomyopathy that results from myocardial deposition of misfolded transthyretin (TTR) protein. The biology of amyloid formation has been elucidated resulting in several effective therapeutic strategies. Accordingly, the therapeutic landscape for ATTR-CM is rapidly evolving, with multiple disease-modifying therapies (DMTs) approved and others anticipated to be imminently available. Currently, DMT strategies involve either stabilization of TTR, thereby inhibiting misfolding, or reduction of hepatic TTR production, and antibodies (“depleters”) that facilitate amyloid fibril removal are under development. In this review, available evidence is synthesized and expert experience provided to assist clinicians in the complex navigation of treatment selection and the role of advanced therapies (heart transplantation and left ventricular assist device), as well as to identify key areas for future research.

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转甲状腺素淀粉样蛋白心肌病的治疗现状

转甲状腺素淀粉样心肌病（atr - cm）是一种浸润性心肌病，由转甲状腺素（TTR）蛋白错误折叠引起的心肌沉积引起。淀粉样蛋白形成的生物学已经被阐明，导致几种有效的治疗策略。因此，atr - cm的治疗前景正在迅速发展，多种疾病修饰疗法（dmt）已获批准，其他疗法预计即将问世。目前，DMT策略包括稳定TTR，从而抑制错误折叠，或减少肝脏TTR的产生，促进淀粉样蛋白纤维去除的抗体（“消耗者”）正在开发中。在这篇综述中，现有的证据是综合和专家经验，以帮助临床医生在治疗选择和先进的治疗方法（心脏移植和左心室辅助装置）的复杂导航，以及确定未来研究的关键领域。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

JACC. Heart failure CARDIAC & CARDIOVASCULAR SYSTEMS-

CiteScore

21.20

自引率

2.30%

发文量

164

期刊介绍： JACC: Heart Failure publishes crucial findings on the pathophysiology, diagnosis, treatment, and care of heart failure patients. The goal is to enhance understanding through timely scientific communication on disease, clinical trials, outcomes, and therapeutic advances. The Journal fosters interdisciplinary connections with neuroscience, pulmonary medicine, nephrology, electrophysiology, and surgery related to heart failure. It also covers articles on pharmacogenetics, biomarkers, and metabolomics.