Abstract Background Heart failure in adult patients with congenital heart disease (ACHD-HF) is prevalent, occurring in 6.4% of the patients. ACHD-HF is associated with an increased mortality risk. Due to the paucity of scientific evidence, specific advice on medical therapies in ACHD-HF and outcome data on contemporary management remains limited. The incidence and significance of different clinical endpoints that could be used in clinical trials is unknown. Purpose Therefore, this study aimed at evaluating (1) (changes in) medical therapy in a contemporary cohort of ACHD-HF patients and (2) the incidence of different (composite) endpoints in different anatomical and pathophysiological subgroups. Methods This retrospective study included 256 ACHD-HF patients (≥16 years), under active follow-up at a tertiary care center, and were followed until latest follow-up visit or until death, ventricular assist device (VAD) placement and/or heart transplant (HTX). Medical therapy was collected at first and last follow-up. Heart failure (HF) diagnosis was defined as signs and/or symptoms of HF requiring medical therapy plus one of the following: (1) impaired ventricular function with elevated intracardiac pressures; (2) elevated BNP or N-terminal BNP; (3) peak oxygen consumption in lowest quartile or (4) unique manifestations in patients with Fontan circulation. Statistical analysis was performed on 200 ACHD-HF patients after exclusion of patients with an early event (1 week of inclusion) or insufficient follow-up. Patients were categorized into mutually exclusive subgroups (figure 1). Endpoints assessed included heart failure hospitalization, diuretics escalation and/or NYHA progression in addition to all-cause mortality, HTX and VAD placement. Results Of the 256 ACHD-HF patients (mean age 50±17 years; 52% male), eventually 200 patients were followed for a median follow-up time of 30 (IQR 22-36) months. Changes in heart failure therapy (panel A) included: increased use of mineralocorticoid receptor antagonists (p=0.038), sodium-glucose transport protein 2 inhibitors (p=0.002) and a trend towards increased use of angiotensin receptor-neprilysin inhibitors (p=0.070). Panel B illustrates the incidence of different (composite) endpoints in different anatomical and pathophysiological subgroups. Furthermore, whereas heart failure hospitalization (p0.001) and NYHA progression (p=0.016) were associated with the occurrence of death, HTX or VAD implant, diuretic escalation (p=0.961) was not (figure 2). Conclusion This observational study illustrates current medical therapy in a contemporary cohort of ACHD-HF patients. In addition to death, HTX and VAD implantation; NYHA progression and heart failure hospitalization are valuable endpoints to define clinical worsening in ACHD patients, which is not the case for diuretics escalation.Figure 1 Figure 2
Bourgeois et al. (Sat,) studied this question.