To evaluate the efficacy and safety of combining targeted pulmonary hypertension drugs with subsequent defect closure (Treat and Repair strategy) in clinical application.

Patients with severe pulmonary hypertension associated with congenital heart disease (CHD-PAH) who had completed right cardiac catheterization (RHC) were retrospectively enrolled in Affiliated Hospital of Xuzhou Medical University from January 2017 to October 2022. All patients were treated with targeted drugs ,and were divided into surgical groups and non-surgical groups according to whether the defect was closed after treatment. Heart function [World Health Organization (WHO)], 6-minite walking distance (6MWD), N-terminal pro-brain natriuretic peptide (NT-proBNP), right atrial diameter, pulmonary artery systolic pressure (echocardiography measurement, PASP_E), pulmonary vascular resistance (PVR), pulmonary artery systolic pressure (RHC measurement, PASP_c), pulmonary flow to systemic flow ratio (Qp/Qs) and other relevant indicators were compared between the two groups, and before and after targeted drug therapy. Logistic regression analysis was used to analyze the related factors affecting whether the patients could close the defect safely. The receiver operating characteristic (ROC) curve was used to analyze the value of each index in predicting whether the patient could safely close the defect.

A total of 41 patients were included, including 16 patients in the surgical group and 25 patients in the non-surgical group. At initial diagnosis, the mean pulmonary artery pressure (mPAP) and PVR in the surgery group were lower than those in the non-surgery group (P=0.011, P<0.001,respectively), and Qp/Qs was higher than that in the non-surgery (P=0.011). After targeted drug therapy, compared with before treatment, there was no statistically significant difference in right ventricular diameter between the two groups (P>0.05). Heart function (WHO) was improved, 6MWD was higher than before treatment, NT-proBNP, right atrial diameter and PASP_E were lower than before treatment (P<0.05). Compared with the initial diagnosis, PVR decreased in the operation group (5.52 WU vs. 4.03 WU, P<0.05) and PVR in the non-operation group (12.80 WU vs. 11.59 WU, P<0.05). Multivariate Logistic regression analysis showed that PVR was an independent predictor of closure after "Treat and Repair"strategy (OR=0.557, 95%CI 0.339-0.914, P=0.020). ROC curve analysis showed that PVR had the largest area under the curve (AUC=0.872, 95%CI 0.762-0.983, P<0.001), truncation value was 7.09, corresponding sensitivity was 75.0%, specificity was 92.1%.

According to previous indications, some patients with severe CHD-PAH who lost the opportunity for surgery could successfully close the defect by using the strategy of "Treat and Repair". Baseline PVR was an independent predictor of successful closure of the defect after "Treat and Repair". The cut-off value of baseline PVR was 7.09 WU. Targeted drugs can improve cardiac function and exercise tolerance in patients with severe CHD-PAH.