# Clinical worsening in adult congenital heart disease and heart failure: A single-centre, observational study

## TL;DR

This study examines treatment trends and markers of worsening in adult patients with congenital heart disease and heart failure.

## Contribution

Identifies NYHA progression and heart failure hospitalization as reliable markers of clinical worsening in ACHD-HF patients.

## Key findings

- Increased use of mineralocorticoid receptor antagonists and sodium-glucose transport protein 2 inhibitors was observed.
- HF hospitalization and NYHA class progression were significantly associated with adverse outcomes like death or VAD implantation.
- Escalation of diuretic therapy was not significantly linked to adverse outcomes.

## Abstract

Data on contemporary treatment approaches and reliable markers of clinical worsening in adult patients with congenital heart disease and heart failure (ACHD-HF) are scarce. This study aimed at evaluating (1) medical therapy within a contemporary ACHD-HF cohort and (2) the incidence of various (composite) endpoints across anatomical and pathophysiological subgroups.

Retrospective study including ACHD-HF patients (≥16 years) under active follow-up at a tertiary care center, monitored until last visit, death, ventricular assist device (VAD) implantation, or heart transplant (HTX). Medical therapy was documented at time of inclusion and final follow-up. Clinical endpoints were evaluated, after excluding patients with early events (<1 week of inclusion) or without follow-up. Endpoints included HF hospitalization, increase of diuretic treatment, NYHA class progression, all-cause mortality, HTX and VAD placement.

Of the 256 ACHD-HF patients (mean age 50 ± 17 years; 52 % male), 56 were excluded for the final analysis. Two hundred patients were followed for a median of 30 (IQR 22–36) months. Changes in medical therapy 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). Furthermore, whereas HF hospitalization (p < 0.001) and progression of NYHA (p = 0.016) were associated with death, whereas HTX or VAD implant and escalation of diuretic therapy (p = 0.961) were not.

This study characterizes current ACHD-HF therapy and identifies NYHA progression and HF hospitalization as potential markers of clinical worsening as they relate to death, HTX, and VAD implantation.

•The incidence of adverse clinical outcomes across distinct anatomical and pathophysiological subgroups of adult patients with congenital heart disease and heart failureremains high.•Both worsening of NYHA functional class, and HF hospitalization were significantly associated with an increased risk of the composite endpoint comprising all-cause mortality, HTX, and/or VAD implant and appear valuable endpoints to assess interventions in ACHD-HF patients.•In contrast, escalation of diuretic therapy did not demonstrate a statistically significant association with adverse outcomes, suggesting limited value as a surrogate endpoint in clinical trials.

The incidence of adverse clinical outcomes across distinct anatomical and pathophysiological subgroups of adult patients with congenital heart disease and heart failureremains high.

Both worsening of NYHA functional class, and HF hospitalization were significantly associated with an increased risk of the composite endpoint comprising all-cause mortality, HTX, and/or VAD implant and appear valuable endpoints to assess interventions in ACHD-HF patients.

In contrast, escalation of diuretic therapy did not demonstrate a statistically significant association with adverse outcomes, suggesting limited value as a surrogate endpoint in clinical trials.