Circulation. 2020 Jul 7;142(1):e7-e22. doi: 10.1161/CIR.0000000000000792. Epub 2020 Jun 1.
Transthyretin amyloid cardiomyopathy (ATTR-CM) results in a restrictive cardiomyopathy caused by extracellular deposition of transthyretin, normally involved in the transportation of the hormone thyroxine and retinol-binding protein, in the myocardium. Enthusiasm about ATTR-CM has grown as a result of 3 simultaneous areas of advancement: Imaging techniques allow accurate noninvasive diagnosis of ATTR-CM without the need for confirmatory endomyocardial biopsies; observational studies indicate that the diagnosis of ATTR-CM may be underrecognized in a significant proportion of patients with heart failure; and on the basis of elucidation of the mechanisms of amyloid formation, therapies are now approved for treatment of ATTR-CM. Because therapy for ATTR-CM may be most effective when administered before significant cardiac dysfunction, early identification of affected individuals with readily available noninvasive tests is essential. This scientific statement is intended to guide clinical practice and to facilitate management conformity by covering current diagnostic and treatment strategies, as well as unmet needs and areas of active investigation in ATTR-CM.
转甲状腺素蛋白淀粉样心肌病（ATTR-CM）是由转甲状腺素蛋白在心肌中外渗沉积引起的限制型心肌病，该蛋白通常参与甲状腺素和视黄醇结合蛋白的转运。由于以下 3 个方面的进展，人们对 ATTR-CM 的兴趣日益浓厚：成像技术可在无需进行有创性心内膜心肌活检的情况下准确地进行非侵入性诊断ATTR-CM；观察性研究表明，在很大一部分心力衰竭患者中，ATTR-CM 的诊断可能被低估；基于对淀粉样形成机制的阐明，目前已有针对 ATTR-CM 的治疗方法获得批准。由于在发生明显的心功能障碍之前进行 ATTR-CM 治疗可能效果最佳，因此使用现有无创检测方法早期识别受影响个体至关重要。本科学声明旨在通过涵盖当前的诊断和治疗策略以及未满足的需求和ATTR-CM 的活跃研究领域，为临床实践提供指导，并促进管理的一致性。
