Department of Pediatrics, University of Miami Miller School of Medicine. Miami FL, USA; Batchelor Children's Research Institute University of Miami Miller School of Medicine. Miami FL, USA.
Interdisciplinary Stem Cell Institute, University of Miami Miller School of Medicine, Miami FL, USA.
Semin Perinatol. 2023 Apr;47(3):151725. doi: 10.1016/j.semperi.2023.151725. Epub 2023 Mar 12.
Congenital heart disease remains one of the most frequently diagnosed congenital diseases of the newborn, with hypoplastic left heart syndrome (HLHS) being considered one of the most severe. This univentricular defect was uniformly fatal until the introduction, 40 years ago, of a complex surgical palliation consisting of multiple staged procedures spanning the first 4 years of the child's life. While survival has improved substantially, particularly in experienced centers, ventricular failure requiring heart transplant and a number of associated morbidities remain ongoing clinical challenges for these patients. Cell-based therapies aimed at boosting ventricular performance are under clinical evaluation as a novel intervention to decrease morbidity associated with surgical palliation. In this review, we will examine the current burden of HLHS and current modalities for treatment, discuss various cells therapies as an intervention while delineating challenges and future directions for this therapy for HLHS and other congenital heart diseases.
先天性心脏病仍然是新生儿最常见的先天性疾病之一,左心发育不全综合征(HLHS)被认为是最严重的一种。在 40 年前,引入了一种复杂的手术姑息治疗方法,包括多个分期手术,跨越了儿童生命的前 4 年,这种单心室缺陷才得以普遍存活。虽然生存率有了显著提高,特别是在有经验的中心,但需要心脏移植的心室衰竭和许多相关的并发症仍然是这些患者持续存在的临床挑战。旨在提高心室功能的基于细胞的治疗方法正在作为一种降低与手术姑息治疗相关的发病率的新干预措施进行临床评估。在这篇综述中,我们将检查 HLHS 的当前负担和当前的治疗方式,讨论各种细胞治疗作为一种干预措施,同时为 HLHS 和其他先天性心脏病的这种治疗方法描绘挑战和未来方向。