转甲状腺素蛋白淀粉样心肌病的当前治疗格局
Current Landscape of Therapies for Transthyretin Amyloid Cardiomyopathy.
作者信息
Griffin Jan M, Grodin Justin L, Ruberg Frederick L, Masri Ahmad, Hanna Mazen, Maurer Mathew S
机构信息
Medical University of South Carolina, Charleston, South Carolina, USA.
University of Southwestern Medical Center, Dallas, Texas, USA.
出版信息
JACC Heart Fail. 2025 May;13(5):685-694. doi: 10.1016/j.jchf.2025.03.017.
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.
转甲状腺素蛋白淀粉样变心肌病（ATTR-CM）是一种浸润性心肌病，由错误折叠的转甲状腺素蛋白（TTR）在心肌中沉积所致。淀粉样蛋白形成的生物学机制已被阐明，从而产生了几种有效的治疗策略。因此，ATTR-CM的治疗格局正在迅速演变，多种疾病修饰疗法（DMT）已获批准，其他疗法预计也即将问世。目前，DMT策略包括稳定TTR以抑制错误折叠，或减少肝脏TTR生成，且促进淀粉样纤维清除的抗体（“清除剂”）正在研发中。在本综述中，我们综合了现有证据并提供了专家经验，以帮助临床医生在复杂的治疗选择过程中做出决策，了解先进疗法（心脏移植和左心室辅助装置）的作用，并确定未来研究的关键领域。
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