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 catheteriza⁃ tion were lower than before（P < 0.05），so was pulmonary artery systolic pressure（PASP）measured by transtho⁃ racic 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. 

Key words:

targeted drug, children, congenital heart disease, pulmonary arterial hypertension, pulmonary vascular reactivity