背景:关于结缔组织病(CTD-PAH)相关肺动脉高压患者的实际临床实践和预后的数据很少。OPUS/OrPHeUS研究招募了新开始Macitentan的患者,包括CTD-PAH.该分析描述了患者特征,治疗模式,结果,使用OPUS/OrPHeUS组合数据集,以及在美国新使用Macitentan的CTD-PAH患者的安全性。
方法:OPUS是前瞻性的,US,多中心,长期的,观察性药物登记(2014年4月至2020年6月)。OrPEUS是一个回顾展,US,多中心医疗图审查(2013年10月-2017年3月)。的特点,治疗模式,安全,和Macitentan治疗CTD-PAH及其系统性硬化症(SSc-PAH)患者的结果,系统性红斑狼疮(SLE-PAH),和混合CTD(MCTD-PAH)与特发性/遗传性PAH(I/HPAH)患者进行描述性比较。
结果:OPUS/OrPEUS合并人群包括2498例I/HPAH患者和1192例CTD-PAH患者(708SSc-PAH;159SLE-PAH;124MCTD-PAH,和201其他CTD-PAH病因)。在I/HPAH和CTD-PAH患者开始接受Macitentan治疗时,分别为:61.2%和69.3%的患者属于世界卫生组织功能类(WHOFC)III/IV;中位6分钟步行距离为289和279m;58.1%和65.2%的患者接受了Macitentan作为联合治疗.随访期间,对于I/HPAH和CTD-PAH患者,分别为:观察到的马西坦暴露的中位持续时间为14.0和15.8个月;79.0和83.0%出现不良事件;Kaplan-Meier估计(95%置信限[CL])在1年时无全因住院的患者分别为60.3%(58.1,62.4)和59.3%(56.1,62.3);Kaplan-Meier估计(95%CL)在1年时的生存率分别为9
结论:Macitentan用于CTD-PAH及其亚组患者的临床实践,包括联合治疗。Macitentan在CTD-PAH患者中的安全性和耐受性与I/HPAH患者相当。
背景:OPSumit®用户注册表(OPUS):NCT02126943;Opsumit®历史用户队列(OrPHeUS):NCT03197688;www.
结果:gov可用于本文的图形摘要。
BACKGROUND: Data on real-world clinical practice and outcomes of patients with pulmonary arterial hypertension associated with connective tissue disease (CTD-PAH) are scarce. The OPUS/OrPHeUS studies enrolled patients newly initiating macitentan, including those with CTD-PAH. This analysis describes patient characteristics, treatment patterns, outcomes, and safety profiles of patients with CTD-PAH newly initiating macitentan in the US using the OPUS/OrPHeUS combined dataset.
METHODS: OPUS was a prospective, US, multicenter, long-term, observational drug registry (April 2014-June 2020). OrPHeUS was a retrospective, US, multicenter medical chart review (October 2013-March 2017). The characteristics, treatment patterns, safety, and outcomes during macitentan treatment of patients with CTD-PAH and its subgroups systemic sclerosis (SSc-PAH), systemic lupus erythematosus (SLE-PAH), and mixed CTD (MCTD-PAH) were descriptively compared to patients with idiopathic/heritable PAH (I/HPAH).
RESULTS: The combined OPUS/OrPHeUS population included 2498 patients with I/HPAH and 1192 patients with CTD-PAH (708 SSc-PAH; 159 SLE-PAH; 124 MCTD-PAH, and 201 other CTD-PAH etiologies). At macitentan initiation for patients with I/HPAH and CTD-PAH, respectively: 61.2 and 69.3% were in World Health Organization functional class (WHO FC) III/IV; median 6-min walk distance was 289 and 279 m; and 58.1 and 65.2% received macitentan as combination therapy. During follow-up, for patients with I/HPAH and CTD-PAH, respectively: median duration of macitentan exposure observed was 14.0 and 15.8 months; 79.0 and 83.0% experienced an adverse event; Kaplan-Meier estimates (95% confidence limits [CL]) of patients free from all-cause hospitalization at 1 year were 60.3% (58.1, 62.4) and 59.3% (56.1, 62.3); and Kaplan-Meier estimates (95% CL) of survival at 1 year were 90.5% (89.1, 91.7) and 90.6% (88.6, 92.3).
CONCLUSIONS: Macitentan was used in clinical practice in patients with CTD-PAH and its subgroups, including as combination therapy. The safety and tolerability profile of macitentan in patients with CTD-PAH was comparable to that of patients with I/HPAH.
BACKGROUND: OPsumit® Users Registry (OPUS): NCT02126943; Opsumit® Historical Users cohort (OrPHeUS): NCT03197688; www.
RESULTS: gov Graphical abstract available for this article.