Safety and Efficacy of Tafamidis in Chinese Patients with Transthyretin Amyloid Cardiomyopathy.

IF 3 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS

Cardiology and Therapy Pub Date : 2025-05-23 DOI:10.1007/s40119-025-00408-6

Zhuang Tian, Daoquan Peng, Wei Ma, Jiangtao Yan, Jian'an Wang, Yida Tang, Wei Jin, Ying Liu, Caiping Jia, Yingxu Gao, Yankun Gong, Xiaohong Sun, Naihan Chen, Shuiqing Zhu, Shuyang Zhang

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引用次数: 0

Abstract

Introduction: Tafamidis is approved in many countries for the treatment of patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Approval is largely based on findings from an international phase 3 trial. This post-approval commitment study aimed to evaluate the safety and efficacy of tafamidis in patients with ATTR-CM in China.

Methods: A multicenter, single-arm study in Chinese patients with symptomatic ATTR-CM in China. All patients received once-daily, open-label tafamidis free acid 61 mg for 12 months. Safety reporting was ongoing with efficacy assessments at months 6 and 12, including 6-min walk test distance, New York Heart Association (NYHA) functional classification, National Amyloidosis Centre staging, N-terminal pro-B-type natriuretic peptide and troponin I concentrations, Kansas City Cardiomyopathy Questionnaire Overall Summary score, 5-level EQ-5D index score, EQ visual analog scale, and 12-item Short Form Survey.

Results: Patients (n = 53) were aged 60 (standard deviation [SD]: 12) years, 89% were male, and 94% had variant ATTR-CM (21% had A97S [p.A117S]). At baseline, most (81%) patients had NYHA class II symptoms (6% class I; 13% class III) and National Amyloidosis Centre stage I disease (74%; 21% stage II; 6% stage III). Median treatment exposure was 345 (range, 24‒418) days. Overall, 85% of patients reported treatment-emergent adverse events (TEAEs). The nature and incidence of TEAEs were consistent with the known safety profile of tafamidis. There were no serious or severe treatment-related TEAEs. At 6 and 12 months, there were minimal changes from baseline in all efficacy outcomes with tafamidis, and a high proportion of patients (≥ 44%) showed clinically relevant stability or improvement in each measure.

Conclusions: The safety of tafamidis in Chinese patients with ATTR-CM was consistent with that previously determined. Tafamidis treatment was associated with a stable disease profile over 12 months in a population of patients where most had variant ATTR-CM and mild heart failure symptoms.

Trial registration: NCT04814186.

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他法非地治疗甲状腺素转淀粉样心肌病的安全性和有效性。

Tafamidis在许多国家被批准用于治疗转甲状腺素淀粉样心肌病（atr - cm）。批准主要基于国际三期试验的结果。这项批准后承诺性研究旨在评估他非他胺在中国ATTR-CM患者中的安全性和有效性。方法：对中国症状性atr - cm患者进行多中心单臂研究。所有患者接受每日一次，开放标签的他非他酸61毫克，持续12个月。安全性报告持续进行，并在第6个月和第12个月进行疗效评估，包括6分钟步行测试距离、纽约心脏协会（NYHA）功能分类、国家淀粉样变性中心分期、n端前b型利钠肽和肌钙蛋白I浓度、堪萨斯城心肌病问卷总体总结评分、5级EQ- 5d指数评分、EQ视觉模拟量表和12项简短调查。结果：53例患者年龄60岁（标准差[SD]: 12）， 89%为男性，94%为变异型atr - cm（21%为A97S [p.A117S]）。在基线时，大多数（81%）患者有NYHA II级症状(6%为I级；13% III类)和国家淀粉样变性中心I期疾病(74%；21%为II期；6% III期)。治疗暴露的中位数为345天（范围24-418天）。总体而言，85%的患者报告了治疗后出现的不良事件（teae）。teae的性质和发生率与已知的tafamidis的安全性一致。没有严重或严重的治疗相关teae。在6个月和12个月时，他法非地的所有疗效结果与基线相比变化很小，并且高比例的患者（≥44%）在每项测量中显示出临床相关的稳定性或改善。结论：中国atr - cm患者使用他非他胺的安全性与先前的研究结果一致。在大多数患有变异性atr - cm和轻度心力衰竭症状的患者人群中，他法非地治疗与超过12个月的稳定疾病概况相关。试验注册：NCT04814186。

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来源期刊

Cardiology and Therapy CARDIAC & CARDIOVASCULAR SYSTEMS-

CiteScore

5.30

自引率

0.00%

发文量

审稿时长

6 weeks

期刊介绍： Aims and Scope Cardiology and Therapy is an international, open access, peer reviewed (single-blind), rapid-publication journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of cardiovascular therapies and interventions, including devices. Studies relating to diagnosis and diagnostics, pharmacoeconomics, public health, quality of life, as well as patient care, management and education are also encouraged. Areas of focus include, but are not limited to, ischaemic heart disease and acute cardiac care, myocardial, valvular, pericardial and congenital heart disease, vascular and pulmonary disease (including hypertension), arrhythmias, heart failure, non-invasive diagnostic techniques, and invasive and interventional cardiology as well as cardiovascular surgery. The journal is of interest to a broad audience of pharmaceutical and healthcare professionals and publishes original research, reviews, case reports/case series, trial protocols and short communications such as commentaries and editorials. Cardiolology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of quality research, which may be considered of insufficient interest by other journals. Rapid Publication The journal’s publication timelines aim for a rapid peer review of 2 weeks. If an article is accepted it will be published 3–4 weeks from acceptance. The rapid timelines are achieved through the combination of a dedicated in-house editorial team, who manage article workflow, and an extensive Editorial and Advisory Board who assist with peer review. This allows the journal to support the rapid dissemination of research, whilst still providing robust peer review. Combined with the journal’s open access model this allows for the rapid, efficient communication of the latest research and reviews, fostering the advancement of cardiovascular therapies. Personal Service The journal’s dedicated in-house editorial team offer a personal “concierge service” meaning authors will always have an editorial contact able to update them on the status of their manuscript. The editorial team check all manuscripts to ensure that articles conform to the most recent COPE, GPP and ICMJE publishing guidelines. This supports the publication of ethically sound and transparent research. Digital Features and Plain Language Summaries Cardiology and Therapy offers a range of additional features designed to increase the visibility, readership and educational value of the journal’s content. Each article is accompanied by key summary points, giving a time-efficient overview of the content to a wide readership. Articles may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand the scientific content and overall implications of the article. The journal also provides the option to include various types of digital features including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations. All additional features are peer reviewed to the same high standard as the article itself. If you consider that your paper would benefit from the inclusion of a digital feature, please let us know. Our editorial team are able to create high-quality slide decks and infographics in-house, and video abstracts through our partner Research Square, and would be happy to assist in any way we can. For further information about digital features, please contact the journal editor (see ‘Contact the Journal’ for email address), and see the ‘Guidelines for digital features and plain language summaries’ document under ‘Submission guidelines’. For examples of digital features please visit our showcase page https://springerhealthcare.com/expertise/publishing-digital-features/ Publication Fees Upon acceptance of your article for publication, authors will be required to pay the mandatory Rapid Service Fee of £3650/€4500/$5100. The journal will consider fee discounts for developing countries and this is decided on a case by case basis. Open Access All articles published by Cardiology and Therapy are published open access. Peer Review Process Upon submission, manuscripts are assessed by the editorial team to ensure they fit within the aims and scope of the journal and are also checked for plagiarism. All suitable submissions are then subject to a comprehensive single-blind peer review. Reviewers are selected based on their relevant expertise and publication history in the subject area. The journal has an extensive pool of editorial and advisory board members who have been selected to assist with peer review based on the afore-mentioned criteria. At least two extensive reviews are required to make the editorial decision, with the exception of some article types such as Commentaries, Editorials and Letters which are generally reviewed by one member of the Editorial Board. Where reviewer recommendations are conflicted, the editorial board will be contacted for further advice and a presiding decision. Manuscripts are then either accepted, rejected or authors are required to make major or minor revisions (both reviewer comments and editorial comments may need to be addressed). Once a revised manuscript is re-submitted, it is assessed along with the responses to reviewer comments and if it has been adequately revised it will be accepted for publication. Accepted manuscripts are then copyedited and typeset by the production team before online publication. Appeals against decisions following peer review are considered on a case by case basis and should be sent to the journal editor. Preprints We encourage posting of preprints of primary research manuscripts on preprint servers, authors’ or institutional websites, and open communications between researchers whether on community preprint servers or preprint commenting platforms. Posting of preprints is not considered prior publication and will not jeopardize consideration in our journals. 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