Clinical Pharmacy Specialist - Cardiology, West Palm Beach Veterans Affairs Medical Center, 7305 N. Military Trial, West Palm Beach, FL, 33410, USA.
PGY2 Cardiology Pharmacy Practice Resident, West Palm Beach Veterans Affairs Medical Center, West Palm Beach, FL, USA.
Curr Cardiol Rep. 2020 Aug 8;22(10):106. doi: 10.1007/s11886-020-01374-2.
To provide a functional review for practicing clinicians on the current and emerging treatment considerations for transthyretin (TTR) cardiac amyloidosis (ATTR-CA).
Current treatment considerations are characterized as those silencing TTR translation, stabilizing TTR tetramers, and disrupting amyloid fibril deposition. Historically considered a rare disease state, ATTR-CA is increasingly recognized as an important mediator of heart failure morbidity and mortality. The emergence of widely available therapies for ATTR-CA has developed hope for patients where little was previously present. Thus, it is important that all cardiology clinicians have a functional understanding of the disease state and treatment options. This review will discuss agents within each of the above classes with expanded discussion on tafamidis given its favorable efficacy, safety, and availability. ATTR-CA diagnostic considerations are reviewed with regard to the identification of potential tafamidis candidates, and practical economic considerations are also reviewed.
为临床医生提供有关转甲状腺素蛋白（TTR）心脏淀粉样变（ATTR-CA）的当前和新兴治疗考虑因素的功能综述。
目前的治疗考虑因素的特点是沉默 TTR 翻译、稳定 TTR 四聚体和破坏淀粉样纤维沉积。ATTR-CA 过去被认为是一种罕见的疾病状态，但现在越来越被认为是心力衰竭发病率和死亡率的一个重要中介。广泛可用的 ATTR-CA 治疗方法的出现为以前几乎没有希望的患者带来了希望。因此，所有心脏病学临床医生都需要对疾病状态和治疗选择有一个功能上的理解。本综述将讨论上述每一类中的药物，并扩展讨论由于其良好的疗效、安全性和可用性而备受关注的他法米替尼。还讨论了针对潜在他法米替尼候选者的 ATTR-CA 诊断考虑因素，以及实际的经济考虑因素。
