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转甲状腺素蛋白淀粉样变心肌病的当前及未来治疗前景

Current and Future Treatment Landscape of Transthyretin Amyloid Cardiomyopathy.

作者信息

Margolin Emily, Stern Lily K, Argiro Alessia, Rosenthal Julie L, Urey Marcus A, Alexander Kevin M

机构信息

Department of Medicine, Division of Cardiovascular Diseases, University of California San Diego, San Diego, CA, USA.

Department of Cardiology, Cedars-Sinai Medical Center, Smidt Heart Institute, Los Angeles, CA, USA.

出版信息

Cardiol Ther. 2025 Jul 19. doi: 10.1007/s40119-025-00424-6.

Abstract

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive disease caused by the deposition of insoluble amyloid fibrils derived from misfolded transthyretin (TTR). The treatment landscape is rapidly evolving, with disease-modifying therapies now targeting distinct steps in disease progression. Management requires both disease-modifying treatment and symptom-guided treatment of heart failure and arrhythmias, along with device therapy and consideration of advanced heart failure interventions (i.e., heart transplantation) in select patients. Therapeutic advances have significantly increased treatment possibilities, selection of appropriate therapy, switching between therapies, combination strategies, and how to monitor treatment response over time. This review summarizes available and investigational therapies for ATTR-CM and considers practical questions that guide clinical decision-making, with the goal of helping clinicians navigate the evolving therapeutic landscape.

摘要

转甲状腺素蛋白淀粉样变心肌病(ATTR-CM)是一种由错误折叠的转甲状腺素蛋白(TTR)衍生的不溶性淀粉样纤维沉积引起的进行性疾病。治疗格局正在迅速演变,疾病修饰疗法现在针对疾病进展的不同阶段。管理既需要疾病修饰治疗,也需要对心力衰竭和心律失常进行症状导向治疗,同时还需要器械治疗,并考虑对特定患者进行晚期心力衰竭干预(即心脏移植)。治疗进展显著增加了治疗可能性、合适疗法的选择、不同疗法之间的转换、联合策略以及如何长期监测治疗反应。本综述总结了ATTR-CM的现有和研究性疗法,并考虑了指导临床决策的实际问题,旨在帮助临床医生应对不断演变的治疗格局。

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