Department of Medicine, Tufts Medical Center, MA, 02111, Boston, USA.
Cardiac Amyloidosis Program, Tufts Medical Center, 800 Washington St., MA, 02111, Boston, USA.
Curr Oncol Rep. 2023 Jun;25(6):549-558. doi: 10.1007/s11912-023-01397-2. Epub 2023 Mar 21.
This review provides an overview of the available therapies for treating neuropathic and/or cardiac manifestations of transthyretin amyloidosis (ATTR), as well as investigational therapeutic agents in ongoing clinical trials. We discuss additional emergent approaches towards thwarting this life-threatening disease that until recently was considered virtually untreatable.
Advances in noninvasive diagnostic methods for detecting ATTR have facilitated easier diagnosis and detection at an earlier stage of disease when therapeutic interventions are likely to be more effective. There are now several ATTR-directed treatments that are clinically available, as well as investigational agents that are being studied in clinical trials. Therapeutic strategies include tetramer stabilization, gene silencing, and fibril disruption. ATTR has been historically underdiagnosed. With advances in diagnostic methods and the advent of disease-modifying treatments, early diagnosis and initiation of treatment is revolutionizing management of this disease.
本篇综述提供了治疗转甲状腺素蛋白淀粉样变性（ATTR）相关神经病和/或心脏表现的现有疗法概述，以及正在进行临床试验的研究性治疗药物。我们讨论了阻止这种危及生命的疾病的其他新方法，因为直到最近，这种疾病还被认为几乎无法治疗。
用于检测ATTR 的非侵入性诊断方法的进步促进了更容易的诊断和早期检测，因为在疾病的早期阶段进行治疗干预可能更有效。目前有几种针对ATTR 的治疗方法已经上市，还有一些研究性药物正在临床试验中进行研究。治疗策略包括四聚体稳定、基因沉默和纤维破坏。ATTR 以前的诊断率较低。随着诊断方法的进步和疾病修饰治疗的出现，早期诊断和治疗的开展正在彻底改变这种疾病的管理。
