Epidemiology and management of patients with pulmonary arterial hypertension associated with congenital heart disease: data from the hellenic pulmonary hypertension registry (HOPE)

Abstract

Abstract Background Pulmonary arterial hypertension (PAH) is a common complication among adult patients with congenital heart disease (CHD). Since clinical trials on PAH-CHD are scarce, registry data are required to shed light on the epidemiology and management of this subgroup. Purpose To present "real-world" data about the epidemiology, management and long-term course of PAH-CHD from the Hellenic Pulmonary Hypertension Registry (HOPE). Methods The HOPE registry was launched in early 2015 and enrols patients from all pulmonary hypertension subgroups in Greece. This analysis focuses on PAH-CHD. Results In total, 85 PAH-CHD patients of whom 50 women (59%) have been enrolled from 8 centers between 2015 and 2022. A follow-up visit was available in 39 patients (45.8%). Median age at first visit was 37 (24, 60) years. About three quarters of patients had a pre-tricuspid (n=32, 37.6%) or a post-tricuspid shunt (n=30, 35.2%), with a quarter of patients having a complex CHD (n=21, 24.7%). Eisenmenger physiology was present in 31 patients (36.4%). The most common comorbidities at baseline were obesity (n=14, 16%), thyroid disease (n=12, 14%) and atrial fibrillation (n=12, 14%). About two thirds of patients were mildly symptomatic at baseline (NYHA I/II, n=55, 64.7%) and remained so at follow-up (n=25/39, 63.7%) with a median time of last available visit at follow-up of 1484 days (4.1 years). According to the three strata ESC risk stratification score, half of patients were at intermediate risk at baseline, while more than half of patients were at low risk for 1-year mortality at follow up (Figure 1A). More than half of patients received PAH monotherapy at baseline (n=46, 54%), with one fifth not receiving any PAH-targeted therapy (n=18, 21.2%). At 4.1 years, half of patients were on combination therapy (n=20/39, 51.3%) (Figure 1B). During a median follow up time of 2547 days (7 years), 17 deaths were reported (0.01 deaths/ patient year) (Figure 2A). 1-, 3- and 5- year survival rates were 96.4% (92.4%-100%), 88.9% (82.3%-96%), and 88.9% (82.3%-96%), respectively. Survival was better among low-risk patients at baseline, compared to moderate and high-risk ones (Figure 2B). Conclusion In this national cohort of ACHD PAH there was a tendency towards combination therapy at follow-up which might be the cause for reduced mortality risk. The three strata ESC risk stratification score may be a useful tool to risk assess these patients for long-term survival.Risk stratification and pharmacotherapyOveral and risk-estimated mortality