Evaluating the safety and long-term efficacy of triple vasodilator therapy for pulmonary arterial hypertension associated with congenital heart disease

Abstract

Abstract Background Pulmonary arterial hypertension (PAH) affects 5-10% of congenital heart disease (CHD) patients, but there is limited information on the safety and efficacy of triple combination therapy for PAH-CHD. The aim of this study was to assess the long-term effects of triple therapy (TT) with prostacyclins on PAH-CHD patients. Methods A retrospective, longitudinal, cohort study of patients with PAH-CHD under active follow-up in our center. All patients were receiving baseline dual therapy at maximum doses. Clinical characteristics, including functional class (FC), 6-minute walk test distance (6MWTd), and NT-ProBNP were recorded before initiating TT and during annual follow-ups during 2 years. Results 60 patients were included (median age 41, 31 women 61%). 32 had Eisenmenger syndrome, 9 had coincidental shunts, 18 had postoperative PAH, and 1 had a significant L-R shunt. TT was initiated based on the clinical situation, existence of shunt and ease of administration (epoprostenol 20%, treprostinil 41%, selexipag 32%, iloprost 7%). A significant improvement in the 6MWTd was observed one year after the initiation of TT (p<0.05), which was maintained at two-year follow-up, with an average improvement of 44 metres. An improvement of FC was observed in 79% of patients at one year, maintained at 2 years in 76%. A NT- proBNP levels decreased from the start, and reached significancy two years after the initiation of TT, with an average decrease of 199 ng/l (Figure 1). Twenty patients (33.3%) experienced side effects after drug initiation. Most were mild and well-controlled with symptomatic treatment. Only 3 (5%) had to change the administration route (Figure 2). By subgroups, patients without Eisenmenger and with pre-tricuspid defects had a more marked benefit. Conclusions TT has been shown to be safe and effective in patients with PAH-CHD, improving FC, 6MWTd, and NT-proBNP levels, particularly in pre-tricuspid defects and non-Eisenmenger PAH-CHD.Changes after initiation of TTType of treatment and side-effects