Julia Vogel, Alexander Carpinteiro, Peter Luedike, Florian Buehning, Simon Wernhart, Tienush Rassaf, Lars Michel
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引用次数: 0
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.
综述的目的:心脏淀粉样变性(CA)是一种以蛋白质错误折叠和细胞外沉积为特征的疾病,可导致器官功能障碍。虽然存在多种形式的 CA,但有两种亚型在临床流行中占主导地位:最近的发现:目前的科学现状反映了推进治疗干预的紧迫性,有 100 多项临床试验正在进行中。心力衰竭的治疗受到 CA 表型的影响,患者对其他常用药物的耐受性较差。治疗合并症(包括心房颤动和瓣膜病)仍是CA的一项挑战,原因是技术困难和结果不确定。Tafamidis 是首个获得批准的 ATTR 稳定剂,其临床使用率正在快速增长。与此同时,各种新的治疗类别也已进入后期临床试验阶段,包括沉默剂、抗体和基因疗法。管理 CA 是未来心衰治疗的关键挑战。本综述描述了当前 CA 治疗的护理标准和科学前景。
期刊介绍:
This journal intends to provide clear, insightful, balanced contributions by international experts that review the most important, recently published clinical findings related to the diagnosis, treatment, management, and prevention of heart failure. We accomplish this aim by appointing international authorities to serve as Section Editors in key subject areas, such as investigative, pharmacologic, and nonpharmacologic therapies, pathophysiology, and prevention. Section Editors, in turn, select topics for which leading experts contribute comprehensive review articles that emphasize new developments and recently published papers of major importance, highlighted by annotated reference lists. An international Editorial Board reviews the annual table of contents, suggests articles of special interest to their country/region, and ensures that topics are current and include emerging research. Commentaries from well-known figures in the field are also provided.