Krasuski Richard A, Tobore Tobore, Studer Sean, Jansa Pavel, Sitbon Olivier, Hoeper Marius M, Channick Richard, Gaine Sean, Lang Irene, Chin Kelly, Pulido Tomas, Mehta Sanjay, Torbicki Adam, Sastry Bhagavatula, Tang Xiaoqin, McLaughlin Vallerie, Reardon Leigh C
Duke University Health System, Durham, North Carolina, USA.
Johnson & Johnson, Titusville, New Jersey, USA.
JACC Adv. 2025 Mar;4(3):101626. doi: 10.1016/j.jacadv.2025.101626. Epub 2025 Feb 24.
Pulmonary arterial hypertension (PAH) is a common complication among patients with congenital heart disease (CHD). Despite advances in PAH treatment, evidence for the benefits of PAH therapies in CHD-PAH is limited.
This analysis aimed to evaluate outcomes in patients with repaired PAH-CHD receiving an approved PAH drug.
This was a pooled analysis including CHD-PAH patients whose CHD was repaired ≥1 year prior from 3 randomized, placebo-controlled, event-driven studies: GRIPHON (NCT01106014), SERAPHIN (NCT00660179), and COMPASS-2 (NCT00303459). The primary endpoint was time to first confirmed morbidity/mortality (M/M) event. HRs with 95% CIs were determined with random effects models.
The analysis included 1,982 patients with PAH, 177 (8.9%) with CHD-PAH. In the overall PAH cohort, the mean age was 48 and 49 years in treatment and placebo groups; 80% and 77% were female. In the CHD-PAH cohort, the mean age was 41 and 39 years; 70% and 66% were female. Overall, ≥98% in each group were World Health Organization functional class II and III at baseline. There was a significant reduction in risk of M/M events vs placebo in the overall PAH and CHD-PAH cohorts: 37% reduction in the overall PAH cohort (HR: 0.63; 95% CI: 0.52-0.77) and 50% reduction in the CHD-PAH population (HR: 0.50; 95% CI: 0.26-0.94).
Treatment with approved PAH drugs provided a similar reduction in M/M risk in patients with repaired CHD-PAH when compared with the overall PAH population. This pooled analysis provides important evidence to guide medical management in this patient population.
肺动脉高压（PAH）是先天性心脏病（CHD）患者的常见并发症。尽管PAH治疗取得了进展，但PAH疗法对CHD-PAH患者益处的证据有限。
本分析旨在评估接受已获批PAH药物治疗的CHD-PAH修复患者的预后。
这是一项汇总分析，纳入了来自3项随机、安慰剂对照、事件驱动研究（GRIPHON（NCT01106014）、SERAPHIN（NCT00660179）和COMPASS-2（NCT00303459））中CHD在≥1年前已修复的CHD-PAH患者。主要终点是首次确认的发病/死亡（M/M）事件的时间。采用随机效应模型确定95%置信区间的风险比（HR）。
分析纳入了1982例PAH患者，其中177例（8.9%）为CHD-PAH患者。在整个PAH队列中，治疗组和安慰剂组的平均年龄分别为48岁和49岁；女性分别占80%和77%。在CHD-PAH队列中，平均年龄分别为41岁和39岁；女性分别占70%和66%。总体而言，每组中≥98%的患者在基线时为世界卫生组织功能分级II级和III级。与安慰剂相比，整个PAH队列和CHD-PAH队列中M/M事件的风险均显著降低：整个PAH队列降低37%（HR：0.63；95%CI：0.52-0.77），CHD-PAH人群降低50%（HR：0.50；95%CI：0.26-0.94）。
与整个PAH人群相比，使用已获批的PAH药物治疗可使CHD-PAH修复患者的M/M风险降低程度相似。这项汇总分析为指导该患者群体的医疗管理提供了重要证据。
