From the 1st Cardiology Department, Center of Cardiomyopathies and Inherited Cardiac Diseases, AHEPA University Hospital, Thessaloniki, Greece.
Cardiol Rev. 2023;31(1):36-41. doi: 10.1097/CRD.0000000000000427. Epub 2021 Oct 20.
Wild-type TTR amyloidosis (wtATTR) represents a disease difficult to diagnose with poor prognosis. Increased clinical suspicion is key, allowing for timely diagnosis. Until recently, only off-label therapies were available but recent introduction of disease specific therapy has shown potential to alter the natural history of the disease. Tafamidis, the only currently approved drug for the therapy of wtATTR, provided significantly better survival and quality of life. However, not all subgroups of patients derived equal benefit. This, along with the increased cost of treatment raised question on whether treatment should be invariably administered through the wtATTR population. This review aims to summarize current evidence on the natural history and staging systems for wtATTR, as well as available treatment options. Special consideration is given to the selection process of patients who would be expected to gain maximum benefit from tafamidis treatment, based on an ethical and cost-effective point of view.
野生型转甲状腺素淀粉样变性（wtATTR）是一种难以诊断且预后不良的疾病。提高临床警惕性是关键，可以实现及时诊断。直到最近，只有超适应证治疗方法可用，但最近引入的疾病特异性治疗方法显示出改变疾病自然史的潜力。特立氟胺是目前唯一获准用于 wtATTR 治疗的药物，显著提高了生存率和生活质量。然而，并非所有亚组患者都能获得同等益处。此外，治疗费用的增加引发了关于是否应普遍向 wtATTR 人群提供治疗的问题。本综述旨在总结 wtATTR 的自然史和分期系统以及现有治疗选择的当前证据。特别考虑了从伦理和成本效益的角度来看，预计从特立氟胺治疗中获益最大的患者的选择过程。
