Int J Cardio：波生坦可治疗唐氏综合征患者的先天性心脏病相关肺动脉高压

口服波生坦对先天性心脏病（CHD）相关的肺动脉高压（PAH）治疗有效，但对唐氏综合征患者的疗效仍然未知。那不勒斯第二大学心血管学院的Michele D’Alto等人对此进行了研究，发现波生坦对于患有唐氏综合征的CHD相关PAH患者同样有效，研究结果发表于《国际心脏病学杂志》。

本次研究的对象为有无唐氏综合征的CHD相关PAH患者，旨在评估波生坦在这两类患者中的长期疗效。对患者基线水平和波生坦治疗12个月后检测发现，两类患者的WHO心功能分级、静息状态下氧饱和度、六分钟步行测试（6MWT）和血液动力学。

研究招募了78名连续病人，18名唐氏综合征患者，另56名非唐氏综合征患者。波生坦治疗12个月后，两组患者心功能分级（唐氏：2.5±0.5 vs 2.9±0.6，p=0.005；对照：2.5±0.5 vs 2.9±0.5，p=0.000002）、六分钟步行测试（唐氏：288±71 vs 239±74 m，p=0.0007；对照：389±80 vs 343±86 m，p=0.00003）和血液动力学（肺动脉血流 唐氏：4.0±1.6 vs 3.5±1.4 l/m/m2，p=0.006；对照：3.5±1.4 vs 2.8±1.0 l/m/m2，p=0.0005；肺动脉/全身血流 唐氏：1.4±0.7 vs 1.0±0.4，p=0.003；对照：1.1±0.7 vs 0.9±0.3，p=0.012；肺循环阻力指数 唐氏：15±9 vs 20±13 WU m2，p=0.007；对照：20±10 vs 26±15 WU m2，p=0.002）均有改善。两治疗组之间疗效没有显著差别。

12个月治疗发现，波生坦对有无唐氏综合征的CHD相关PAH患者安全性和耐受性良好。无论有无唐氏综合征，患者的临床状态、运动耐受和肺动脉血液动力学均有改善。

Pulmonary arterial hypertension associated with congenital heart disease.
Abstract
Pulmonary arterial hypertension associated with congenital heart disease, although common (15%-30%) in all-comers with congenital heart disease, is variable in terms of clinical manifestations, severity of associated pulmonary arterial hypertension, and response to therapy and outcomes (depending on lesion anatomy, pulmonary circulation flows and pressures, and presence and timings of surgeries). Evaluation includes imaging and catheterization. Surgical or another anatomic correction may be desirable after rigorous preinterventional assessment. Patients who are not surgical candidates or who fail to improve early or late after surgery may have the potential to respond to idiopathic pulmonary arterial hypertension therapies. Lung or heart/lung transplantation remains an option for selected recalcitrant patients.Pulmonary arterial hypertension associated with congenital heart disease, although common (15%-30%) in all-comers with congenital heart disease, is variable in terms of clinical manifestations, severity of associated pulmonary arterial hypertension, and response to therapy and outcomes (depending on lesion anatomy, pulmonary circulation flows and pressures, and presence and timings of surgeries). Evaluation includes imaging and catheterization. Surgical or another anatomic correction may be desirable after rigorous preinterventional assessment. Patients who are not surgical candidates or who fail to improve early or late after surgery may have the potential to respond to idiopathic pulmonary arterial hypertension therapies. Lung or heart/lung transplantation remains an option for selected recalcitrant patients.