【摘要】 目的 评价靶向药物对儿童先天性心脏病（CHD）合并肺动脉高压（PAH）治疗效果，评估靶向药物对这些患儿肺血管反应性的治疗效果。方法 前瞻分析2017年1月至2021年3月在本中心经心导管检查确诊为中重度PAH的CHD患儿，予靶向药物治疗至少3个月后再次心导管检查，随访观察临床效果。第一次心导管时11例行急性肺血管反应试验（AVT）。结果 共15例患儿，靶向药物治疗后经心导管检查的肺动脉平均压（mPAP）、肺血管阻力指数（PVRI）以及经胸超声心动图测得肺动脉收缩压（PASP）较治疗前明显下降（P <0.05）。8例（53.3%）经靶向药物治疗后行手术治疗，余7例继续口服靶向药物治疗。所有行AVT试验的患儿中，仅18.2%(2/11)提示阳性。结论 靶向药物可降低CHD合并PAH患儿的mPAP及PVRI，甚至可以获得解剖根治手术机会。先心病患儿的AVT试验阳性率低，靶向药物治疗可以作为一种新的评估肺血管反应性的标准。更多还原

【Abstract】 Objective To evaluate the efficacy of targeted drugs for congenital heart disease(CHD)with pulmonary arterial hypertension(PAH)in children,and to evaluate the role of targeted drugs in the diagnosis and treatment of pulmonary vascular reactivity. Methods A prospective analysis was performed on the children with CHD diagnosed as moderate to severe PAH by cardiac catheterization from January 2017 to March 2021. The patients received targeted drug therapy for at least three months and then underwent repeated cardiac catheterization. Acute vasoreactivity testing(AVT)was performed in 11 patients at the first cardiac catheterization. Results 15 children were enrolled in this study. After treatment with targeted drugs,mPAP and PVRI measured via cardiac catheterization were lower than before(P < 0.05),so was pulmonary artery systolic pressure(PASP)measured by transthoracic echocardiography. Eight patients(53.3%)received surgical treatment after drug therapy,while the rest seven continued drug therapy. AVT was positive only in 18.2%(2/11)of all the children. Conclusions Targeted drugs can reduce both mPAP and PVRI in children with CHD complicated by PAH,and even offer the children an option of anatomic radical surgery. For a low positive rate of AVT test in children with CHD,targeted drug therapy can be used as a new standard to assess pulmonary vascular reactivity.更多还原