转甲状腺素淀粉样心肌病:推进精准医学的范例。

IF 37.6 1区 医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS
Esther Gonzalez-Lopez, Mathew S Maurer, Pablo Garcia-Pavia
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引用次数: 0

摘要

针对潜在疾病机制的特定疗法的发展构成了精准医学的基础。转甲状腺素型心脏淀粉样变性(atr - cm)是心衰和心肌病领域精确治疗方法的典范。更好地了解潜在的病理生理学,更精确的流行病学数据,以及允许非侵入性诊断的成像技术的进步,促进了atr - cm新的和非常有效的特异性治疗的发展。治疗的进步已经彻底改变了这个领域,把一种罕见的、毁灭性的、无法治愈的疾病变成了一种更常见的疾病,有几种治疗方法可供选择。三种主要类型的治疗方法(稳定剂,抑制剂和降解剂)作用于淀粉样蛋白级联的不同点,已经开发或目前正在研究中。本文综述了近几十年来atr - cm在病理生理学和流行病学方面的主要进展,以及现有或正在开发的不同治疗方案,说明了精准医学在心血管疾病中的作用。还审查了未来几年需要回答的未决问题。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Transthyretin amyloid cardiomyopathy: a paradigm for advancing precision medicine.

Development of specific therapies addressing the underlying diseases' mechanisms constitutes the basis of precision medicine. Transthyretin cardiac amyloidosis (ATTR-CM) is an exemplar of precise therapeutic approach in the field of heart failure and cardiomyopathies. A better understanding of the underlying pathophysiology, more precise data of its epidemiology, and advances in imaging techniques that allow non-invasive diagnosis have fostered the development of new and very effective specific therapies for ATTR-CM. Therapeutic advances have revolutionized the field, transforming a rare, devastating, and untreatable disease into a more common disease with several therapeutic alternatives available. Three main types of therapies (stabilizers, suppressors, and degraders) that act at different points of the amyloidogenic cascade have been developed or are currently under investigation. In this review, the key advances in pathophysiology and epidemiology that have occurred in the last decades along with the different therapeutic alternatives available or under development for ATTR-CM are described, illustrating the role of precision medicine applied to cardiovascular disorders. Pending questions that would need to be answered in upcoming years are also reviewed.

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来源期刊
European Heart Journal
European Heart Journal 医学-心血管系统
CiteScore
39.30
自引率
6.90%
发文量
3942
审稿时长
1 months
期刊介绍: The European Heart Journal is a renowned international journal that focuses on cardiovascular medicine. It is published weekly and is the official journal of the European Society of Cardiology. This peer-reviewed journal is committed to publishing high-quality clinical and scientific material pertaining to all aspects of cardiovascular medicine. It covers a diverse range of topics including research findings, technical evaluations, and reviews. Moreover, the journal serves as a platform for the exchange of information and discussions on various aspects of cardiovascular medicine, including educational matters. In addition to original papers on cardiovascular medicine and surgery, the European Heart Journal also presents reviews, clinical perspectives, ESC Guidelines, and editorial articles that highlight recent advancements in cardiology. Additionally, the journal actively encourages readers to share their thoughts and opinions through correspondence.
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