Rosenthal Lisa-Maria, Miera Oliver, Krauss Annemarie, Danne Friederike, Berger Felix, Kramer Peter
Department of Congenital Heart Disease - Pediatric Cardiology, Deutsches Herzzentrum der Charité (DHZC), Berlin, Germany.
Charité Universitätsmedizin Berlin, Corporate Member of Freie Universität and Humboldt-Universität zu Berlin, Berlin, Germany.
ESC Heart Fail. 2025 Jul 21. doi: 10.1002/ehf2.15386.
Dapagliflozin, a sodium-glucose cotransporter 2 (SGLT2) inhibitor, has shown clinical benefits in adults with heart failure (HF), improving cardiac function, reducing HF-related hospitalizations and enhancing survival rates. While extensively studied in adult HF, data on its efficacy and safety in paediatric HF patients remain limited. We aimed to evaluate the use of dapagliflozin in addition to optimized therapy in paediatric HF patients regarding safety, clinical outcomes and adverse events.
We conducted a single-centre retrospective analysis of 37 paediatric HF patients (median age 9.0 years, range 0.2-17.1 years) treated with dapagliflozin at our institution between April 2022 and February 2025. Clinical outcomes, left ventricular ejection fraction (LVEF), global longitudinal strain (GLS), NT-proBNP levels and estimated glomerular filtration rate (eGFR) were analysed at baseline, 3-6 months and the latest follow-up. The most frequent diagnoses among paediatric HF patients treated with dapagliflozin were dilated cardiomyopathy (43.2%, 56% of those with acute myocarditis), heart transplant recipients (18.9%) and single ventricle heart defects (16.2%). The median duration of dapagliflozin treatment was 189 days (Q1, Q3: 381, 596). Dapagliflozin was well tolerated, with no severe adverse effects observed. During follow-up, four patients required ventricular assist device (VAD) implantation, five underwent heart transplantation and one patient died. In six patients, the VAD could be explanted due to myocardial recovery. Overall, LVEF significantly improved from 40% at baseline to 51% at 3-6 months and further to 57% at latest follow-up (P = 0.016). GLS significantly improved from -9.2% to -14.7% from baseline to latest follow-up (P = 0.023). Heart failure classification significantly improved from baseline to latest follow-up (P = 0.004). NT-proBNP levels decreased during follow-up, without reaching statistical significance.
Dapagliflozin in addition to optimized HF therapy was safe and well tolerated in paediatric HF patients, with improvements in functional class, left ventricular contractility and heart failure symptoms. The study's limitations, including its small sample size and retrospective design, highlight the need for larger, multicentre, prospective trials to confirm these findings.
达格列净是一种钠-葡萄糖协同转运蛋白2（SGLT2）抑制剂，已在成年心力衰竭（HF）患者中显示出临床益处，可改善心脏功能、减少与HF相关的住院次数并提高生存率。虽然在成年HF患者中进行了广泛研究，但其在儿科HF患者中的疗效和安全性数据仍然有限。我们旨在评估在优化治疗基础上加用达格列净对儿科HF患者的安全性、临床结局和不良事件的影响。
我们对2022年4月至2025年2月期间在我院接受达格列净治疗的37例儿科HF患者（中位年龄9.0岁，范围0.2 - 17.1岁）进行了单中心回顾性分析。在基线、3 - 6个月和最新随访时分析临床结局、左心室射血分数（LVEF）、整体纵向应变（GLS）、N末端脑钠肽前体（NT-proBNP）水平和估计肾小球滤过率（eGFR）。接受达格列净治疗的儿科HF患者中最常见的诊断为扩张型心肌病（43.2%，急性心肌炎患者中的56%）、心脏移植受者（18.9%）和单心室心脏缺陷（16.2%）。达格列净治疗的中位持续时间为189天（第一四分位数、第三四分位数：381，596）。达格列净耐受性良好，未观察到严重不良反应。在随访期间，4例患者需要植入心室辅助装置（VAD），5例接受了心脏移植，1例患者死亡。6例患者因心肌恢复可移除VAD。总体而言，LVEF从基线时的40%显著改善至3 - 6个月时的51%，并在最新随访时进一步提高至57%（P = 0.016）。从基线到最新随访，GLS从 - 9.2%显著改善至 - 14.7%（P = 0.023）。从基线到最新随访，心力衰竭分级显著改善（P = 0.004）。随访期间NT-proBNP水平下降，但未达到统计学意义。
在优化HF治疗基础上加用达格列净对儿科HF患者安全且耐受性良好，功能分级、左心室收缩力和心力衰竭症状均有改善。该研究的局限性，包括样本量小和回顾性设计，突出了需要进行更大规模、多中心、前瞻性试验来证实这些发现。
