Pediatric Advanced Cardiac Care and Transplantation, Division of Pediatric Cardiology, Columbia University Vagelos College of Physicians and Surgeons, New York, New York, USA.
Curr Opin Pediatr. 2024 Oct 1;36(5):489-495. doi: 10.1097/MOP.0000000000001393. Epub 2024 Aug 12.
This article highlights the most recent advances in a review of the current literature in the field of pediatric heart failure and transplantation.
Diagnostically, the identification of new genetic factors has contributed to a deeper understanding of cardiomyopathy in children. Novel medications like sacubitril/valsartan and Sodium-Glucose cotransporter-2 (SGLT2) inhibitors, which are now standard in the adult population are being studied in pediatric population and offer new promise of pediatric heart failure treatment. Ventricular assist devices are more commonly used in cardiomyopathy patients and single ventricle patients as a bridge to transplant. Recent pediatric heart transplant society (PHTS) data demonstrated that waitlist survival improved significantly over the past decades (i) and new treatments such as daratumumab and eculizumab have been used in high-risk populations and demonstrate promising results. TEAMMATE trial is the first multicenter randomized clinical trial (RCT) in pediatric heart transplant (HT) to evaluate the safety and efficacy of everolimus (EVL) and low-dose tacrolimus (TAC) compared to standard-dose TAC and mycophenolate mofetil (MMF). It will provide valuable information about the safety and efficacy of EVL, TAC, and MMF (ii).Donor cell-free DNA has been used more in pediatric transplant recipients and has significantly decreased invasive EMB (iii).
This past 5 years have witness dramatic progress in the field of pediatric heart failure and transplantation including more use of mechanical support in heart failure patients with various underlying etiology, especially use of mechanical support in single ventricle patients and the use of sacubitril/valsartan and SGLT2 inhibitors in the pediatric population. The problem of the highly sensitized transplant recipient remains, although novel therapeutics have been added to our toolbox of options to maintain healthy allograft function. Ongoing research aims to further enhance our understanding and management of pediatric heart failure, emphasizing the need for continued innovation in this complex field.
本文重点介绍了儿科心力衰竭和移植领域文献综述的最新进展。
在诊断方面,新的遗传因素的鉴定有助于深入了解儿童心肌病。新型药物,如沙库巴曲缬沙坦和钠-葡萄糖共转运蛋白 2(SGLT2)抑制剂,目前在成人中已成为标准治疗方法,也正在儿科人群中进行研究,为儿科心力衰竭的治疗提供了新的希望。心室辅助装置在心肌病患者和单心室患者中更常用于作为移植前的过渡。最近的儿科心脏移植协会(PHTS)数据表明,过去几十年,等待名单上的存活率显著提高(i),并且新的治疗方法,如达鲁单抗和依库珠单抗,已在高危人群中使用,并取得了有希望的结果。TEAMMATE 试验是第一个评估依维莫司(EVL)和低剂量他克莫司(TAC)与标准剂量 TAC 和霉酚酸酯(MMF)相比在儿科心脏移植(HT)中的安全性和疗效的多中心随机临床试验(RCT)(ii)。它将提供有关 EVL、TAC 和 MMF 的安全性和疗效的宝贵信息(ii)。无细胞供体 DNA 已在儿科移植受者中得到更广泛的应用,并显著降低了侵入性 EMB(iii)。
过去 5 年,儿科心力衰竭和移植领域取得了显著进展,包括在各种潜在病因的心力衰竭患者中更广泛地使用机械支持,尤其是在单心室患者中使用机械支持以及在儿科人群中使用沙库巴曲缬沙坦和 SGLT2 抑制剂。高度致敏的移植受者仍然存在问题,尽管我们的治疗选择工具箱中增加了新的治疗方法,但仍需要保持健康的同种异体移植物功能。正在进行的研究旨在进一步提高我们对儿科心力衰竭的认识和管理水平,强调在这个复杂领域需要不断创新。