Section of Cardiology, Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO.
Section of Cardiology, Department of Pediatrics, Columbia University Irving Medical Center, Morgan Stanley Childrens Hospital of New York Presbyterian Hospital, New York, NY.
J Pediatr. 2024 Dec;275:114221. doi: 10.1016/j.jpeds.2024.114221. Epub 2024 Aug 2.
To describe the safety and effectiveness of treating pediatric patients who have pulmonary arterial hypertension (PAH) with selexipag in a real-world, multicenter cohort, given that data supporting its use in pediatric PAH are sparse.
We report a multicenter, retrospective, cohort study of children with PAH treated with selexipag. Demographic and clinical variables were extracted from the medical records. Clinical parameters were analyzed at 3 timepoints: before selexipag, 3-12 months after selexipag, and >12 months follow-up.
Eighty-seven patients were included, 32 received selexipag as add-on to background therapy, and 55 transitioned from another prostanoid. The median starting and final doses were 4.7 and 28.5 μg/kg/dose twice daily, respectively. Add-on patients demonstrated improved indexed pulmonary to systemic vascular resistance ratio after selexipag initiation (PVRi/SVRi, 0.62v0.53; P = .034) with a lower average mean pulmonary artery pressure (46 vs 39 mm Hg; P = NS), and oxygen consumption (maximal oxygen consumption during cardiopulmonary exercise testing [VO max] 27.8 mL/kg/min vs 30.9 mL/kg/min; P = NS). Transition patients demonstrated stable mean pulmonary artery pressure (47 mm Hg vs 45 mm Hg; P = NS) and a lower mean indexed pulmonary vascular resistance (10.9 Wood units∗m vs 8.2 Wood units∗m; P = NS) but late functional worsening in some with VO max decreased at follow-up (26.0 mL/kg/min vs 19.5 mL/kg/min). Side effects were noted in 40% of the cohort, but prompted discontinuation in only 2%.
In a large, multicenter cohort, the oral prostacyclin agonist selexipag demonstrates favorable tolerability and effectiveness. Add-on patients demonstrated early hemodynamic improvement. Transition patients demonstrated early stability with risk of late functional worsening, highlighting the importance of ongoing monitoring.
描述在真实世界多中心队列中，使用塞来昔帕治疗患有肺动脉高压（PAH）的儿科患者的安全性和有效性，因为支持其在儿科 PAH 中应用的数据很少。
我们报告了一项多中心、回顾性、队列研究，纳入了接受塞来昔帕治疗的 PAH 患儿。从病历中提取人口统计学和临床变量。在 3 个时间点分析临床参数：塞来昔帕治疗前、塞来昔帕治疗后 3-12 个月和随访>12 个月。
共纳入 87 例患儿，32 例患儿接受塞来昔帕作为背景治疗的附加治疗，55 例患儿从其他前列环素类药物转换而来。起始和最终剂量中位数分别为 4.7μg/kg/剂量，每日 2 次和 28.5μg/kg/剂量，每日 2 次。附加治疗组患者在开始塞来昔帕治疗后，肺血管阻力指数/体循环血管阻力指数（PVRi/SVRi）改善（0.62 比 0.53；P=0.034），平均肺动脉压（mPAP）（46 比 39mmHg；P=NS）和最大耗氧量（心肺运动试验时最大耗氧量[VO2max]，27.8 比 30.9mL/kg/min；P=NS）均降低。转换治疗组患者 mPAP 稳定（47mmHg 比 45mmHg；P=NS），平均肺血管阻力指数（10.9 Wood 单位m 比 8.2 Wood 单位m；P=NS）较低，但一些患者在随访时 VO2max 下降，表现为晚期功能恶化（26.0 比 19.5mL/kg/min）。该队列中有 40%的患儿出现不良反应，但仅有 2%的患儿因不良反应而停药。
在一项大型多中心队列中，口服前列环素激动剂塞来昔帕具有良好的耐受性和有效性。附加治疗组患者早期血流动力学改善。转换治疗组患者早期稳定，但有晚期功能恶化的风险，这突出了持续监测的重要性。
