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当前心脏淀粉样变性治疗的疗法和未来展望。

Current Therapies and Future Horizons in Cardiac Amyloidosis Treatment.

机构信息

Department of Cardiology and Vascular Medicine, West German Heart and Vascular Center, University Hospital Essen, Hufelandstraße 55, 45147, Essen, Germany.

Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, Hufelandstraße 55, 45147, Essen, Germany.

出版信息

Curr Heart Fail Rep. 2024 Aug;21(4):305-321. doi: 10.1007/s11897-024-00669-7. Epub 2024 May 29.

Abstract

PURPOSE OF REVIEW

Cardiac amyloidosis (CA) is a condition characterized by misfolding and extracellular deposition of proteins, leading to organ dysfunction. While numerous forms of CA exist, two subtypes dominate clinical prevalence: Transthyretin amyloid (ATTR) and immunoglobulin light chain amyloid.

RECENT FINDINGS

The current scientific landscape reflects the urgency to advance therapeutic interventions with over 100 ongoing clinical trials. Heart failure treatment is affected by CA phenotype with poor tolerance of otherwise frequently used medications. Treating comorbidities including atrial fibrillation and valvular disease remains a challenge in CA, driven by technical difficulties and uncertain outcomes. Tafamidis is the first ATTR-stabilizer approved with a rapidly growing rate of clinical use. In parallel, various new therapeutic classes are in late-stage clinical trials including silencers, antibodies and genetic therapy. Managing CA is a critical challenge for future heart failure care. This review delineates the current standard-of-care and scientific landscape of CA therapy.

摘要

目的综述

心脏淀粉样变性(CA)是一种由蛋白质错误折叠和细胞外沉积引起的疾病,导致器官功能障碍。虽然存在多种形式的 CA,但两种亚型在临床患病率中占主导地位:转甲状腺素蛋白淀粉样变(ATTR)和免疫球蛋白轻链淀粉样变。

最新发现

目前的科学领域反映出迫切需要推进治疗干预措施,目前正在进行 100 多项临床试验。心力衰竭的治疗受到 CA 表型的影响,对其他常用药物的耐受性差。治疗合并症,包括心房颤动和瓣膜病,在 CA 中仍然是一个挑战,这是由技术困难和不确定的结果驱动的。他司美琼是第一种获批的 ATTR 稳定剂,其临床应用率正在迅速增长。与此同时,各种新的治疗类别正在进行后期临床试验,包括沉默剂、抗体和基因治疗。管理 CA 是未来心力衰竭治疗的一个关键挑战。本综述阐述了 CA 治疗的当前标准护理和科学领域。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/daf6/11333534/ca78f53abf62/11897_2024_669_Fig1_HTML.jpg

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