The effect of long-term tafamidis treatment on quality of life in people with transthyretin amyloid cardiomyopathy (ATTR-CM): A plain language summary.

IF 1.6 Q3 CARDIAC & CARDIOVASCULAR SYSTEMS

Future cardiology Pub Date : 2024-01-01 Epub Date: 2024-09-11 DOI:10.1080/14796678.2024.2391250

Martha Grogan, Margot K Davis, Maria G Crespo Leiro, Marla B Sultan, Balarama Gundapaneni, Franca Stedile Angeli, Mazen Hanna

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引用次数: 0

Abstract

What is this summary about?: This summary explains some results of a study called ATTR-ACT and its ongoing long-term extension study that were published in the European Journal of Heart Failure. The purpose of ATTR-ACT was to find out if a drug called tafamidis is an effective treatment for people with a heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM). People took tafamidis or placebo for up to 2.5 years in ATTR-ACT (the initial study). A placebo looks like the study medicine but does not contain any active ingredients. After people completed the initial study, they could take part in the extension study. An extension study allows people to continue receiving treatment after the original clinical study ends and helps researchers understand how well a treatment works over a longer time period. This extension study allows people to receive tafamidis for up to an additional 5 years. People who took placebo in the initial study now receive tafamidis. People who took tafamidis in the initial study continue tafamidis treatment. Researchers looked at changes in peoples' ability to enjoy life ('quality of life') and heart failure symptoms since they started ATTR-ACT. Results are available for the first 2.5 years of the extension study.

What are the key takeaways?: During the initial study, there was less worsening of quality of life and heart failure symptoms in people who took tafamidis compared to people who took placebo. In the extension study, quality of life and heart failure symptoms were maintained or nearly maintained in people who took tafamidis in the initial study. In people who started tafamidis in the extension study, quality of life and heart failure symptoms continued to worsen, but the worsening slowed down.

What were the main conclusions reported by the researchers?: Tafamidis slows the worsening of quality of life and heart failure symptoms in people with ATTR-CM. People with ATTR-CM should start treatment early to receive the most benefit.Clinical Trial Registration: NCT01994889 (ATTR-ACT) (ClinicalTrials.gov).

查看原文本刊更多论文

经淀粉样蛋白心肌病（ATTR-CM）患者长期服用他伐米迪对生活质量的影响：通俗易懂的摘要。

本摘要介绍了发表在《欧洲心力衰竭杂志》（European Journal of Heart Failure）上的一项名为 ATTR-ACT 的研究及其正在进行的长期扩展研究的部分结果。ATTR-ACT研究的目的是了解一种名为他伐米迪的药物是否能有效治疗一种名为转甲状腺素淀粉样变性心肌病（ATTR-CM）的心脏病患者。在 ATTR-ACT（初始研究）中，患者服用他伐米迪或安慰剂长达 2.5 年。安慰剂看起来与研究药物相似，但不含任何活性成分。完成初始研究后，患者可以参加扩展研究。扩展研究允许患者在最初的临床研究结束后继续接受治疗，有助于研究人员了解治疗在较长时间内的效果。这项延长研究允许患者继续接受塔法米地治疗长达5年。在初始研究中服用安慰剂的患者现在可以服用他法米迪。在初始研究中服用过他法米迪的人继续接受他法米迪治疗。研究人员观察了患者自开始接受 ATTR-ACT 治疗以来在享受生活的能力（"生活质量"）和心衰症状方面的变化。扩展研究的前两年半已经有了结果：在最初的研究中，与服用安慰剂的患者相比，服用他伐米迪的患者生活质量和心衰症状的恶化程度较低。在扩展研究中，在初始研究中服用他法米迪的患者的生活质量和心衰症状得以维持或基本维持。在延伸研究中，开始服用他法米迪的患者的生活质量和心衰症状继续恶化，但恶化速度放缓：研究人员报告的主要结论是什么？ATTR-CM患者应尽早开始治疗，以获得最大疗效：NCT01994889（ATTR-ACT）（ClinicalTrials.gov）。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Future cardiology CARDIAC & CARDIOVASCULAR SYSTEMS-

CiteScore

2.80

自引率

5.90%

发文量

期刊介绍： Research advances have contributed to improved outcomes across all specialties, but the rate of advancement in cardiology has been exceptional. Concurrently, the population of patients with cardiac conditions continues to grow and greater public awareness has increased patients" expectations of new drugs and devices. Future Cardiology (ISSN 1479-6678) reflects this new era of cardiology and highlights the new molecular approach to advancing cardiovascular therapy. Coverage will also reflect the major technological advances in bioengineering in cardiology in terms of advanced and robust devices, miniaturization, imaging, system modeling and information management issues.