治疗心脏淀粉样变性的新疗法。

IF 2 4区 医学 Q3 CARDIAC & CARDIOVASCULAR SYSTEMS
Current Opinion in Cardiology Pub Date : 2025-03-01 Epub Date: 2025-01-13 DOI:10.1097/HCO.0000000000001198
Olives Nguyen, Daniel Kamna, Ahmad Masri
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引用次数: 0

摘要

综述目的:综述转甲状腺素(atr - cm)和免疫球蛋白轻链(AL-CM)心脏淀粉样变性的治疗进展。最近的研究发现:在atr - cm中,他法底斯仍然是基础治疗,美国食品和药物管理局(FDA)批准了5年以上。Acoramidis是另一种促甲状腺素稳定剂,最近在ATTRibute-CM试验中取得积极结果后获得fda批准。Vutrisiran是一种转甲状腺素基因消声器,在HELIOS-B试验中显示出疗效,正在等待FDA的审查。Eplontersen的CARDIO-TTRansform试验是迄今为止最大的atr - cm研究,预计将于2025年底报告。诸如NTLA-2001(一种CRISPR-Cas9疗法)和ALXN2220和coramitug等纤维消耗剂等创新方法正在临床试验中取得进展。在AL-CM中,达拉单抗、环磷酰胺、硼替佐米和地塞米松(Dara-CyBorD)已经确立了自己的治疗标准。新的抗浆细胞疗法包括CAR-T细胞和双特异性抗体(teclistimab)和纤维消耗物。Birtamimab在VITAL试验期间提高了晚期AL-CM的生存率,目前正在对AFFIRM-AL进行研究。Anselamimab正在进行III期临床试验,而AT-02正在进行atr - cm和AL-CM的早期试验。摘要:atr - cm和AL-CM的治疗前景正在迅速发展,受到针对不同机制的新疗法的推动。正在进行的临床试验有望进一步完善治疗标准,改善心脏淀粉样变性患者的预后。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
New therapies to treat cardiac amyloidosis.

Purpose of review: Review advancements in therapies for transthyretin (ATTR-CM) and immunoglobulin light chain (AL-CM) cardiac amyloidosis.

Recent findings: In ATTR-CM, tafamidis remains the cornerstone therapy, with Food and Drug Administration (FDA) approval for over 5 years. Acoramidis, another transthyretin stabilizer, has very recently been FDA-approved following positive results in the ATTRibute-CM trial. Vutrisiran, a transthyretin gene silencer, demonstrated efficacy in the HELIOS-B trial and awaits FDA review. Eplontersen's CARDIO-TTRansform trial, the largest ATTR-CM study to date, is expected to report by late 2025. Innovative approaches such as NTLA-2001 (a CRISPR-Cas9 therapy) and fibril depleters like ALXN2220 and coramitug are advancing in clinical trials. In AL-CM, daratumumab, cyclophosphamide, bortezomib, and dexamethasone (Dara-CyBorD) has established itself as the standard of care. Novel antiplasma cell therapies include CAR-T cells and bispecific antibodies (teclistimab) and fibril depleters. Birtamimab improved survival in advanced AL-CM during the VITAL trial and is under investigation in AFFIRM-AL. Anselamimab is in phase III CARES trials, whereas AT-02 undergoes early-phase testing for ATTR-CM and AL-CM.

Summary: The therapeutic landscape for ATTR-CM and AL-CM is rapidly evolving, driven by novel therapies targeting diverse mechanisms. Ongoing clinical trials promise to further refine the standard of care and improve outcomes for patients with cardiac amyloidosis.

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来源期刊
Current Opinion in Cardiology
Current Opinion in Cardiology 医学-心血管系统
CiteScore
4.20
自引率
4.30%
发文量
78
审稿时长
6-12 weeks
期刊介绍: ​​​​​​Current Opinion in Cardiology is a bimonthly publication offering a unique and wide ranging perspective on the key developments in the field. Each issue features hand-picked review articles from our team of expert editors. With fourteen disciplines published across the year – including arrhythmias, molecular genetics, HDL cholesterol and clinical trials – every issue also contains annotated reference detailing the merits of the most important papers.
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