患有肺动脉高血压的儿童的macitentan:一个多中心体验
A Rodriguez Ogando1,2, A Sabaté-Rotés3, A Moya Bonora4
1Department of Paediatric Cardiology, Health Research Institute Gregorio Marañón, Gregorio Marañon General University Hospital, Madrid, Spain. alejandro.rodriguez@salud.madrid.org.
Pediatric cardiology
|July 5, 2025
概括
在患有肺动脉高血压 (PAH) 的儿科患者中,macitentan治疗显著改善了结果,显示出功能类和运动能力的增强. 该药物在这个年轻人群中显示出良好的安全性和有效性.
科学领域:
- 儿童心脏病学 儿童心脏病学
- 肺高血压研究 肺高血压研究
- 药理学研究 药理学研究
背景情况:
- 儿童肺动脉高血压 (PAH) 需要有效的治疗选择.
- 马西坦是一种内甲素受体对抗剂,在成人PAH中表现有前途,但需要在儿科患者群体中进行评估.
- 关于马西坦在18岁以下患者的安全性和疗效的数据有限.
研究的目的:
- 评估口服马西坦在患有1组PAH的儿科患者的安全性和疗效.
- 评估临床结果,包括功能类,运动能力和生化标志物.
- 为了确定药物的耐受性和对PAH儿童风险分层的影响.
主要方法:
- 采用西班牙儿科肺高血压登记处数据的多中心观察性研究.
- 包括74名患有1组PAH的儿科患者 (平均年龄9.6岁).
- 评估6个月后的结果,包括世界卫生组织功能类 (WHO FC),6分钟步行距离 (6MWD),NT-proBNP,风险参数和生活质量.
主要成果:
- 世卫组织FC显著改善,世卫组织FCIII/IV患者的患者数量从48%降至22% (p=0.001).
- 平均6MWD增加了大约45米 (p=0.032).
- NT-proBNP水平显著下降 (平均为-438.9 pg/ml,p=0.015),符合低风险标准的患者增加了17% (p=0.002).
- 在5.4%的患者中报告了不良事件,其中一例中断了macitentan治疗.
结论:
- 口服macitentan在患有1组PAH的儿科患者中显示出显著的临床益处和良好的耐受性.
- 这些发现支持在常规临床实践中使用macitentan来治疗儿科PAH.
- 进一步的研究可能会探索儿童的长期结果和特定的PAH亚型.
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