一项当代、现实世界的混合表现型转甲状腺蛋白淀粉样心肌病患者的生存率：来自THAOS的分析。
IF 3
Q2 CARDIAC & CARDIOVASCULAR SYSTEMS
引用次数: 0
摘要
Tafamidis被批准用于治疗转甲状腺素淀粉样心肌病（atr - cm）。许多atr - cm患者表现为心脏和神经系统症状的混合表型，但缺乏他非他汀在这一人群中的实际有效性研究。该研究评估了现实世界中接受他非他汀治疗和未接受治疗的混合表型atr - cm患者的生存和其他结果。方法：转甲状腺素淀粉样变性结局调查（THAOS）是一项纵向、观察性的4期研究，研究对象是转甲状腺素淀粉样变性患者和致病性转甲状腺素基因变异的无症状携带者，研究于2023年6月完成。该分析纳入了2019-2023年纳入THAOS的当代患者队列，这些患者在入组时具有混合表型atr - cm。在整个研究过程中，接受他非他胺治疗的队列接受了批准剂量的他非他胺（meglumine 80 mg/free acid 61 mg），而未接受治疗的队列从未接受过他非他胺。结果：在接受他非他汀治疗（n = 116）和未接受他非他汀治疗（n = 223）的患者中，入组时的中位年龄分别为77.8岁和72.8岁，42.2%和77.6%的患者有变异型atr - cm。30个月时，接受他非他汀治疗的患者生存率为81.5% (95% CI 66.7-90.2)，未接受他非他汀治疗的患者生存率为75.1% （95% CI 66.1-82.0）。他非他汀治疗组心血管相关住院的中位年发病率为0.89，未治疗组为1.70，心血管相关住院的中位时间分别为7.0天和11.5天。两组分别有13例（11.2%）和40例（17.9%）死亡。结论：与未经治疗的患者相比，经批准剂量的他非他汀治疗的混合表型atr - cm患者生存率更高，心血管相关住院率更低，死亡率更低。这些数据与最近在THAOS中主要为心脏atr - cm患者的结果相似，并将atr - act的结果扩展到当代，现实世界，混合表型人群。试验注册：ClinicalTrials.gov识别码NCT00628745。本文章由计算机程序翻译，如有差异，请以英文原文为准。
Survival in a Contemporary, Real-World Cohort of Patients with Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis: An Analysis from THAOS.
Introduction: Tafamidis is approved to treat transthyretin amyloid cardiomyopathy (ATTR-CM). Many patients with ATTR-CM present with a mixed phenotype of both cardiac and neurologic symptoms, but real-world effectiveness studies of tafamidis in this population are lacking. This study assessed survival and other outcomes in a real-world, contemporary cohort of tafamidis-treated and untreated patients with mixed-phenotype ATTR-CM.
Methods: The Transthyretin Amyloidosis Outcomes Survey (THAOS) was a longitudinal, observational, phase 4 study of patients with transthyretin amyloidosis and asymptomatic carriers of pathogenic transthyretin gene variants and was completed in June 2023. This analysis included a contemporary cohort of patients enrolled in THAOS in 2019-2023 who were characterized as having mixed-phenotype ATTR-CM at enrollment. The tafamidis-treated cohort received the approved dose of tafamidis (meglumine 80 mg/free acid 61 mg) throughout the study, and the untreated cohort never received tafamidis.
Results: In tafamidis-treated (n = 116) and untreated patients (n = 223), respectively, median age at enrollment was 77.8 and 72.8 years, and 42.2% and 77.6% had variant ATTR-CM. Survival rates at 30 months were 81.5% (95% CI 66.7-90.2) in tafamidis-treated patients and 75.1% (95% CI 66.1-82.0) in untreated patients. Median yearly incidence of cardiovascular-related hospitalizations was 0.89 for tafamidis-treated and 1.70 for untreated patients, and median duration of cardiovascular-related hospitalizations was 7.0 and 11.5 days, respectively. There were 13 (11.2%) and 40 (17.9%) deaths in the respective groups.
Conclusion: Patients with mixed-phenotype ATTR-CM treated with the approved dose of tafamidis had numerically higher survival rates, a numerically lower rate of cardiovascular-related hospitalizations, and fewer deaths than untreated patients. These data parallel recent results for patients with predominantly cardiac ATTR-CM from THAOS and extend results of ATTR-ACT to a contemporary, real-world, mixed-phenotype population.
Trial registration: ClinicalTrials.gov identifier NCT00628745.
求助全文
通过发布文献求助，成功后即可免费获取论文全文。
去求助
来源期刊
Cardiology and Therapy
CARDIAC & CARDIOVASCULAR SYSTEMS-
CiteScore
5.30
自引率
0.00%
发文量
38
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. Authors should disclose details of preprint posting during the submission process or at any other point during consideration in one of our journals. Once the preprint is published, it is the author’s responsibility to ensure that the preprint record is updated with a publication reference, including the DOI and a URL link to the published version of the article on the journal website.
Copyright
Cardiology and Therapy is published under the Creative Commons Attribution-Noncommercial License, which allows users to read, copy, distribute, and make derivative works for non-commercial purposes from the material, as long as the author of the original work is cited. The author assigns the exclusive right to any commercial use of the article to Springer. For more information about the Creative Commons Attribution-Noncommercial License, click here: http://creativecommons.org/licenses/by-nc/4.0.
Contact
For more information about the journal, including pre-submission enquiries, please contact matthew.evans@springer.com
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
