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Abstract：
Cardiac amyloidosis is a restrictive cardiomyopathy that results from the deposition of misfolded light chain or transthyretin proteins, most commonly, in cardiac tissue. Traditionally, treatment options for light chain (AL) and transthyretin (ATTR) amyloidosis have been limited. However, there are now multiple novel therapeutics in development and several therapeutics recently approved that promise to revolutionize clinical management of AL and ATTR. Most of these agents disrupt specific stages of amyloidogenesis such as light chain or transthyretin protein production, formation of amyloidogenic intermediates, or amyloid fibril aggregation. Others aim to remove existing amyloid tissue deposits using monoclonal antibody technology. Although these advances represent an important step forward in the care of cardiac amyloidosis patients, additional studies are needed to define the optimal treatment paradigms for AL and ATTR and to validate clinical, imaging, or serum biomarker strategies that may confirm a cardiac response to therapy.
摘要：
心脏淀粉样变性是一种限制性心肌病，由错误折叠的轻链或甲状腺素运载蛋白的沉积引起，最常见的是,在心脏组织中。传统上,轻链(AL)和甲状腺素运载蛋白(ATTR)淀粉样变性的治疗选择有限.然而,目前有多种新疗法正在开发中,最近批准的几种疗法有望彻底改变AL和ATTR的临床管理.这些药物中的大多数破坏淀粉样蛋白生成的特定阶段，如轻链或运甲状腺素蛋白的产生，淀粉样中间体的形成，或淀粉样蛋白原纤维聚集。其他人的目标是使用单克隆抗体技术去除现有的淀粉样蛋白组织沉积物。尽管这些进展代表了心脏淀粉样变性患者护理的重要一步，需要更多的研究来确定AL和ATTR的最佳治疗模式，并验证临床，成像,或血清生物标志物策略，可以确认心脏对治疗的反应。
