Macitentan in Pulmonary Arterial Hypertension Due to Congenital Heart Disease (CHD-PAH): Real-World Evidence from the OPUS/OrPHeUS Studies.

IF 3 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Cardiology and Therapy Pub Date : 2024-11-25 DOI:10.1007/s40119-024-00386-1
Kelly M Chin, Richard Channick, Nick H Kim, Rose Ong, Stefano Turricchia, Nicolas Martin, Lada Mitchell, Vallerie V McLaughlin
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Abstract

Introduction: Data on real-world clinical practice and outcomes of patients with pulmonary arterial hypertension (PAH) associated with congenital heart disease (CHD) are scarce. The OPUS/OrPHeUS studies enrolled patients with PAH newly initiating macitentan, including those with PAH associated with CHD (CHD-PAH).

Methods: OPUS was a prospective, United States, multicenter, long-term, observational drug registry (April 2014-June 2020). OrPHeUS was a retrospective, United States, multicenter medical chart review (October 2013-March 2017). The characteristics, treatment patterns, safety, and outcomes during macitentan treatment of patients with CHD-PAH and the subgroups Eisenmenger syndrome, left-to-right shunts and small/coincidental CHD were descriptively compared.

Results: The combined OPUS/OrPHeUS population included 272 (6.1%) patients with CHD-PAH (80 patients with Eisenmenger syndrome; 82 patients with left-to-right shunts and 92 patients with small/coincidental defects). Most patients across the CHD-PAH subgroups were in World Health Organization Functional Class II/III (82.9-94.6%). Macitentan was initiated as combination therapy in 65.0% of patients with CHD-PAH. During follow-up, 81.4% of patients experienced ≥ 1 adverse event (AE), the most common being dyspnea (23.5%), nausea (13.7%), dizziness (12.7%), headache (12.7%) and edema (10.8%). The 1- and 2-year Kaplan-Meier (95% confidence limits) estimates of patients with CHD-PAH being free from hospitalization were 64.5% (57.9, 70.4) and 49.3% (41.9, 56.3); for survival, the 1- and 2-year Kaplan-Meier estimates were 93.5% (89.3, 96.1) and 90.2% (84.9, 93.7).

Conclusions: Macitentan was used in clinical practice in patients with CHD-PAH and its subgroups, including as combination therapy in the majority of patients. Safety in this population was consistent with the known profile of macitentan.

Trial registration: OPsumit® Users Registry (OPUS): NCT02126943; Opsumit® Historical Users cohort (OrPHeUS): NCT03197688; URL www.

Clinicaltrials: gov .

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马西替坦治疗先天性心脏病引起的肺动脉高压(CHD-PAH):来自 OPUS/OrPHeUS 研究的真实世界证据。
导言:有关伴有先天性心脏病(CHD)的肺动脉高压(PAH)患者的实际临床实践和疗效的数据很少。OPUS/OrPHeUS 研究招募了新开始使用马西替坦的 PAH 患者,包括伴有先天性心脏病(CHD-PAH)的 PAH 患者:OPUS是一项前瞻性的美国多中心长期观察性药物登记研究(2014年4月至2020年6月)。OrPHeUS是一项回顾性美国多中心病历审查(2013年10月至2017年3月)。对CHD-PAH患者以及艾森曼格综合征、左至右分流和小/偶发性CHD亚组患者在马西替坦治疗期间的特征、治疗模式、安全性和结果进行了描述性比较:OPUS/OrPHeUS合并人群包括272名(6.1%)CHD-PAH患者(80名艾森曼格综合征患者、82名左向右分流患者和92名小/偶发性缺损患者)。大多数 CHD-PAH 亚组患者属于世界卫生组织功能分级 II/III 级(82.9%-94.6%)。65.0%的CHD-PAH患者开始接受马西替坦联合治疗。随访期间,81.4%的患者发生了≥1次不良事件(AE),最常见的不良事件为呼吸困难(23.5%)、恶心(13.7%)、头晕(12.7%)、头痛(12.7%)和水肿(10.8%)。CHD-PAH患者1年和2年免住院的Kaplan-Meier估计值(95%置信区间)分别为64.5%(57.9, 70.4)和49.3%(41.9, 56.3);1年和2年的Kaplan-Meier估计值分别为93.5%(89.3, 96.1)和90.2%(84.9, 93.7):马西替坦可用于CHD-PAH患者及其亚组的临床实践,包括大多数患者的联合治疗。该人群的安全性与马西替坦的已知特征一致:试验注册:OPsumit® 用户注册中心(OPUS):NCT02126943; Opsumit® Historical Users cohort (OrPHeUS):NCT03197688; URL www.Clinicaltrials: gov .
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来源期刊
Cardiology and Therapy
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
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