Advances in understanding and managing pediatric heart failure and transplant.

IF 2.2 3区 医学 Q2 PEDIATRICS Current opinion in pediatrics Pub Date : 2024-08-12 DOI:10.1097/mop.0000000000001393
Wenyuan Xu,Marc Richmond
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Abstract

PURPOSE OF REVIEW This article highlights the most recent advances in a review of the current literature in the field of pediatric heart failure and transplantation. RECENT FINDINGS 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). SUMMARY 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.
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了解和管理小儿心力衰竭和移植方面的进展。
最新发现诊断方面,新遗传因素的确定有助于加深对儿童心肌病的了解。新药物如沙库比曲利/缬沙坦和钠-葡萄糖共转运体-2(SGLT2)抑制剂现已成为成人的标准药物,目前正在儿科人群中进行研究,为儿科心力衰竭治疗带来了新希望。心室辅助装置更常用于心肌病患者和单心室患者,作为移植的桥梁。儿科心脏移植协会(PHTS)的最新数据显示,过去几十年来,候补名单上的存活率显著提高(i),达拉单抗(daratumumab)和依库珠单抗(eculizumab)等新疗法已用于高风险人群,并取得了良好的效果。TEAMMATE 试验是儿科心脏移植(HT)领域的首个多中心随机临床试验(RCT),旨在评估依维莫司(EVL)和小剂量他克莫司(TAC)与标准剂量 TAC 和霉酚酸酯(MMF)相比的安全性和有效性。它将为 EVL、TAC 和 MMF 的安全性和有效性提供有价值的信息(ii)。无捐献者细胞 DNA 已更多地用于儿科移植受者,并显著减少了侵袭性 EMB(iii)。摘要过去五年来,儿科心力衰竭和移植领域取得了巨大进步,包括对有各种潜在病因的心力衰竭患者更多地使用机械支持,尤其是对单心室患者使用机械支持,以及在儿科人群中使用沙库比曲/缬沙坦和 SGLT2 抑制剂。尽管我们的工具箱中增加了新的治疗方法来维持健康的异体移植功能,但高度敏感的移植受体问题依然存在。正在进行的研究旨在进一步加强我们对小儿心力衰竭的了解和管理,强调了在这一复杂领域持续创新的必要性。
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来源期刊
CiteScore
6.20
自引率
0.00%
发文量
184
审稿时长
6-12 weeks
期刊介绍: ​​​​​Current Opinion in Pediatrics is a reader-friendly resource which allows the reader to keep up-to-date with the most important advances in the pediatric field. Each issue of Current Opinion in Pediatrics contains three main sections delivering a diverse and comprehensive cover of all key issues related to pediatrics; including genetics, therapeutics and toxicology, adolescent medicine, neonatology and perinatology, and orthopedics. Unique to Current Opinion in Pediatrics is the office pediatrics section which appears in every issue and covers popular topics such as fever, immunization and ADHD. Current Opinion in Pediatrics is an indispensable journal for the busy clinician, researcher or student.
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