Background: Pulmonary arterial hypertension associated with congenital heart disease (PAH-CHD) is a very heterogeneous disease. There are few published data on the effect of PAH-specific therapy in patients with PAH-CHD and they included only patients with Eisenmenger's syndrome (ES) and PAH after defect correction (DC).
Purpose: The aim of the study was to evaluate the effects of PAH-specific therapy in the 4 clinical subgroups of PAH-CHD patients: ES, PAH associated with prevalent systemic-to-pulmonary shunts, PAH with small/coincidental defects, DC.
Methods: From 1977 to December 2017 consecutive patients with PAH-CHD referred to our centre were included in the study. All patients underwent baseline clinical evaluation, six-minute walking distance (6MWD) and right heart catheterization. The same evaluations were performed before and 3–4 months after the beginning of a new PAH-specific drug initiated in our centre. Changes in 6MWD and haemodynamic parameters were analyzed using Wilcoxon signed-rank test and compared between the 4 clinical subgroups of PAH-CHD patients with Kruskal-Wallis test. Data are presented as median (interquartile range).
Results: 231 consecutive PAH-CHD patients (50% ES, 19% S/P, 6% SD, 25% DC) were enrolled. Patients with complex CHD were excluded from the analysis. Median follow-up was 117 (54–275) months. 102 patients began monotherapy (55 ES, 18 S/P, 7 SD, 22 CS) and 82 patients associated double combination therapy in our centre (44 ES, 10 S/P, 5 SD, 23 CS). Patients who received triple combination therapy were not analyzed because of the small size of the sample. Results are shown in the Table.
Conclusions: Initial monotherapy and double sequential combination therapy were effective in improving haemodynamic profile and exercise capacity in patients with PAH-CHD without any significant difference between the four clinical subgroups.