Initial clinical experience with dapagliflozin in addition to optimized medical therapy in paediatric heart failure patients

IF 3.7 2区医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS

ESC Heart Failure Pub Date : 2025-07-21 DOI:10.1002/ehf2.15386

Lisa-Maria Rosenthal, Oliver Miera, Annemarie Krauss, Friederike Danne, Felix Berger, Peter Kramer

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Abstract

Aims

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.

Methods and results

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.

Conclusions

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.

Abstract Image

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除了优化药物治疗外，达格列净在儿科心力衰竭患者中的初步临床经验

目的：钠-葡萄糖共转运蛋白2 （SGLT2）抑制剂达格列净（Dapagliflozin）在成人心力衰竭（HF）患者中显示出临床益处，可改善心功能，减少HF相关住院，提高生存率。虽然在成人心衰中进行了广泛的研究，但其在儿科心衰患者中的有效性和安全性的数据仍然有限。我们的目的是评估在优化治疗的基础上使用达格列净对小儿心衰患者的安全性、临床结局和不良事件的影响。方法和结果：我们对2022年4月至2025年2月在我院接受达格列净治疗的37例小儿心衰患者（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、左室射血分数（LVEF）、整体纵向应变（GLS）、NT-proBNP水平和肾小球滤过率（eGFR）。在接受达格列净治疗的儿童心衰患者中，最常见的诊断是扩张型心肌病（43.2%，56%的急性心肌炎患者）、心脏移植受体（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（Q1, Q3: 381,596）。达格列净耐受性良好，未观察到严重的不良反应。随访期间，4例患者需要心室辅助装置（VAD）植入，5例患者接受心脏移植，1例患者死亡。6例患者因心肌恢复，可将VAD切除。总体而言，LVEF从基线时的40%显著改善到3-6个月时的51%，在最新随访时进一步改善到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，心衰分类明显改善（P = 0.004）。NT-proBNP水平在随访期间下降，但无统计学意义。结论：在优化心衰治疗的基础上，达格列净在儿童心衰患者中是安全且耐受性良好的，功能分级、左心室收缩力和心衰症状均有改善。该研究的局限性，包括其小样本量和回顾性设计，突出了需要更大的、多中心的、前瞻性的试验来证实这些发现。

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来源期刊

ESC Heart Failure Medicine-Cardiology and Cardiovascular Medicine

CiteScore

7.00

自引率

7.90%

发文量

461

审稿时长

12 weeks

期刊介绍： ESC Heart Failure is the open access journal of the Heart Failure Association of the European Society of Cardiology dedicated to the advancement of knowledge in the field of heart failure. The journal aims to improve the understanding, prevention, investigation and treatment of heart failure. Molecular and cellular biology, pathology, physiology, electrophysiology, pharmacology, as well as the clinical, social and population sciences all form part of the discipline that is heart failure. Accordingly, submission of manuscripts on basic, translational, clinical and population sciences is invited. Original contributions on nursing, care of the elderly, primary care, health economics and other specialist fields related to heart failure are also welcome, as are case reports that highlight interesting aspects of heart failure care and treatment.