Chin Kelly M, Channick Richard, Kim Nick H, Ong Rose, Turricchia Stefano, Martin Nicolas, Mitchell Lada, McLaughlin Vallerie V
UT Southwestern Medical Center, 5939 Harry Hines Blvd, Dallas, TX, 75390, USA.
University of California Los Angeles, Los Angeles, CA, USA.
Cardiol Ther. 2024 Dec;13(4):775-796. doi: 10.1007/s40119-024-00386-1. Epub 2024 Nov 25.
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).
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.
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).
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.
OPsumit Users Registry (OPUS): NCT02126943; Opsumit Historical Users cohort (OrPHeUS): NCT03197688; URL www.
gov .
关于先天性心脏病（CHD）相关肺动脉高压（PAH）患者的真实世界临床实践和结局的数据稀缺。OPUS/OrPHeUS研究纳入了新开始使用马昔腾坦的PAH患者，包括CHD相关PAH（CHD-PAH）患者。
OPUS是一项前瞻性、美国多中心、长期观察性药物注册研究（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研究（2013年10月至2017年3月）。对CHD-PAH患者以及艾森曼格综合征、左向右分流和小/偶发性CHD亚组患者在使用马昔腾坦治疗期间的特征、治疗模式、安全性和结局进行了描述性比较。
OPUS/OrPHeUS联合人群包括272例（6.1%）CHD-PAH患者（80例艾森曼格综合征患者；82例左向右分流患者和92例小/偶发性缺陷患者）。CHD-PAH亚组中的大多数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患者开始使用马昔腾坦作为联合治疗。在随访期间，81.�%的患者发生≥1次不良事件（AE），最常见的是呼吸困难（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年无住院的Kaplan-Meier（95%置信区间）估计值分别为64.5%（57.9，70.4）和49.3%（41.9，56.3）；对于生存率，1年和两年的Kaplan-Meier估计值分别为93.5%（89.3，96.1）和90.2%（84.9，93.7）。
马昔腾坦用于CHD-PAH患者及其亚组的临床实践中，包括大多数患者作为联合治疗。该人群中的安全性与马昔腾坦已知的特征一致。
OPsumit用户注册研究（OPUS）：NCT02126943；Opsumit历史用户队列（OrPHeUS）：NCT03197688；网址www.CLINICALTRIALS.gov 。
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