达格列净治疗丰坦治疗失败的成人患者（DAPA-FONTAN）：一项前瞻性多中心研究

IF 2.2 3区医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS

Archives of Cardiovascular Diseases Pub Date : 2025-09-01 DOI:10.1016/j.acvd.2025.06.009

Mathieu Albertini , Victor Waldmann , Valentin Baland , Elise Barre , Jelena Radojevic , Laurianne Le Gloan , Patrice Guérin , Fabien Labombarda , Fanny Dion , Reaksmei Ly , François Godart , Pamela Moceri , Pauline David , Alexis Barat , Antoine Legendre , Anne-Solene Chaussade , Sebastien Hascoet , Laurence Iserin

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引用次数: 0

摘要

心力衰竭（HF）是成人先天性心脏病患者死亡的主要原因，特别是单室心脏和Fontan循环患者。随着时间的推移，这种循环与发病率增加有关，特别是在循环衰竭的情况下（Fontan-FF衰竭）。FF有多种原因：单心室收缩或舒张功能障碍、肺血管阻力升高、明显的瓣膜返流或Fontan回路狭窄。治疗选择是有限的，目前推荐用于左侧hf的达格列净可能在这一特定人群中提供功能性益处。目的评价达格列净对收缩期或舒张期功能障碍的成年FF患者功能容量的影响。方法采用6个月的前瞻性、多中心、观察性研究。纳入标准包括过去6个月内经右心导管确诊的18岁以上FF患者，定义为：-收缩功能障碍，单室射血分数(EF)≤40%，心室舒张末期压（VEDP）或肺动脉楔压(PAWP)≥15 mmHg。-舒张功能障碍，EF≥41%，VEDP或paap≥15mmhg。排除标准包括严重房室瓣返流、肺血管阻力升高、丰坦回路狭窄、妊娠、eGFR和lt 25 ml/min/1.73 m2、收缩压和lt 95 mmHg。主要终点：基线至6个月期间心肺运动试验（CPET）最大摄氧量的变化。次要终点：NYHA分级、生活质量（KCCQ-12）、NT-proBNP水平、EF、hf相关住院次数、利尿剂剂量、肝肾功能、血压和不良事件。预期结果预期患者的运动能力、NYHA评分、生活质量和生物学参数均有改善，治疗具有良好的临床和生物学耐受性。该研究可能支持在有收缩期或舒张期功能障碍的FF患者中靶向使用达格列净，并为未来在这一高危人群中的随机试验奠定基础。

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Dapagliflozin in Adult Patients with Failing Fontan (DAPA-FONTAN): A Prospective Multicentre Study

Introduction

Heart failure (HF) is the leading cause of death in adults with congenital heart disease, particularly in patients with a univentricular heart and Fontan circulation. Over time, this circulation is associated with increasing morbidity, especially in cases of circulatory failure (Failing Fontan–FF). FF has multiple causes: systolic or diastolic dysfunction of the single ventricle, elevated pulmonary vascular resistance, significant valve regurgitation, or stenoses within the Fontan circuit. Therapeutic options are limited, and Dapagliflozin—currently recommended for left-sided HF—may offer functional benefits in this specific population.

Objective

To assess the impact of Dapagliflozin on functional capacity in adult patients with FF due to systolic or diastolic dysfunction.

Methods

A 6-month prospective, multicenter, observational study.Inclusion criteria include adults older than 18 years with FF confirmed by right heart catheterization within the past 6 months, defined by:

– Systolic dysfunction with single ventricular ejection fraction (EF) ≤ 40% and ventricular end-diastolic pressure (VEDP) or pulmonary artery wedge pressure (PAWP) ≥ 15 mmHg.

– Diastolic dysfunction with EF ≥ 41% and VEDP or PAWP ≥ 15 mmHg.Exclusion criteria include severe atrioventricular valve regurgitation, elevated pulmonary vascular resistance, Fontan circuit stenosis, pregnancy, eGFR < 25 ml/min/1.73 m², and systolic BP < 95 mmHg.Primary endpoint: Change in VO₂ max on cardiopulmonary exercise testing (CPET) between baseline and 6 months.Secondary endpoints: NYHA class, quality of life (KCCQ-12), NT-proBNP levels, EF, number of HF-related hospitalizations, diuretic dosage, liver and kidney function, blood pressure, and adverse events.

Expected Results

An improvement in exercise capacity, NYHA class, quality of life, and biological parameters is expected, with good clinical and biological tolerance of the treatment.

Perspectives

This study may support the targeted use of Dapagliflozin in FF patients with systolic or diastolic dysfunction and lay the groundwork for future randomized trials in this high-risk population.

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来源期刊

Archives of Cardiovascular Diseases 医学-心血管系统

CiteScore

4.40

自引率

6.70%

发文量

审稿时长

34 days

期刊介绍： The Journal publishes original peer-reviewed clinical and research articles, epidemiological studies, new methodological clinical approaches, review articles and editorials. Topics covered include coronary artery and valve diseases, interventional and pediatric cardiology, cardiovascular surgery, cardiomyopathy and heart failure, arrhythmias and stimulation, cardiovascular imaging, vascular medicine and hypertension, epidemiology and risk factors, and large multicenter studies. Archives of Cardiovascular Diseases also publishes abstracts of papers presented at the annual sessions of the Journées Européennes de la Société Française de Cardiologie and the guidelines edited by the French Society of Cardiology.