Evolution of Disease-modifying Therapy for Transthyretin Cardiac Amyloidosis.

IF 1.9 Q3 CARDIAC & CARDIOVASCULAR SYSTEMS Heart International Pub Date : 2024-06-27 eCollection Date: 2024-01-01 DOI:10.17925/HI.2024.18.1.5
Adam Ioannou
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Abstract

Transthyretin cardiac amyloidosis (ATTR-CA) represents an inexorably progressive and fatal cardiomyopathy. Increased understanding of the underlying pathogenesis responsible for the misfolding of transthyretin and the subsequent accumulation of amyloid fibrils within the myocardium has led to the development of several disease-modifying therapies that act on different stages of the disease pathway. Tafamidis is the first, and to date remains the only, therapy approved for the treatment of ATTR-CA, which, alongside acoramidis, stabilizes the transthyretin tetramer, preventing disaggregation, misfolding and formation of amyloid fibrils. Gene-silencing agents, such as patisiran, vutrisian and eplontersen, and novel gene-editing therapies, such as NTLA-2001, act to reduce the hepatic synthesis of transthyretin. Anti-amyloid therapies represent another strategy in the treatment of ATTR-CA and are designed to bind amyloid fibril epitopes and stimulate macrophage-mediated removal of amyloid fibrils from the myocardium. Many of these treatments are at an early investigational stage but represent an important area of unmet clinical need and could potentially reverse disease and restore cardiac functions even in patients with advanced disease.

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转甲状腺素心脏淀粉样变性疾病的疾病修饰疗法的演变。
转甲状腺素心脏淀粉样变性(ATTR-CA)是一种不可避免的进展性致命心肌病。随着人们对导致转甲状腺素错误折叠以及随后淀粉样纤维在心肌内聚集的潜在发病机制认识的加深,开发出了多种针对疾病不同阶段的治疗方法。Tafamidis是第一种,也是迄今为止唯一一种获准用于治疗ATTR-CA的疗法,它与阿考拉米迪一起稳定转甲状腺素四聚体,防止其分解、错误折叠和淀粉样纤维的形成。帕替西兰(Patisiran)、武曲先(Vutrisian)和易普隆特生(Eplontersen)等基因沉默剂以及 NTLA-2001 等新型基因编辑疗法可减少转甲状腺素在肝脏的合成。抗淀粉样蛋白疗法是治疗 ATTR-CA 的另一种策略,旨在结合淀粉样蛋白纤维表位,刺激巨噬细胞介导的淀粉样蛋白纤维从心肌中清除。其中许多治疗方法还处于早期研究阶段,但代表着一个尚未满足临床需求的重要领域,即使是晚期患者也有可能逆转病情并恢复心脏功能。
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来源期刊
Heart International
Heart International Medicine-Cardiology and Cardiovascular Medicine
CiteScore
0.90
自引率
0.00%
发文量
9
审稿时长
7 weeks
期刊最新文献
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