Raissadati Alireza, Tolani Drishti, Profita Elizabeth L
Department of Pediatrics (Cardiology), Stanford University, Palo Alto, California.
Department of Pediatrics (Cardiology), Cleveland Clinic, Cleveland, Ohio.
JHLT Open. 2025 Aug 7;10:100360. doi: 10.1016/j.jhlto.2025.100360. eCollection 2025 Nov.
Pediatric cardiomyopathy (CMP), with an incidence of approximately 1 in 100,000 children, remains a critical clinical challenge. Often progressive, these myocardial disorders frequently culminate in end-stage heart failure, establishing CMP as the primary indication for pediatric heart transplantation. Key considerations for transplantation include specific CMP etiologies and subtypes alongside established listing criteria and strategies to navigate waitlist complexities. The evolving role of mechanical circulatory support, especially ventricular assist devices (VADs), has transformed bridging strategies and significantly improved survival to transplant. Contemporary post-transplant outcomes demonstrate continued improvement, with advancements in survival rates and ongoing refinement of essential long-term management strategies. This review provides a comprehensive analysis of pediatric CMP as it progresses to end-stage heart failure requiring transplantation. It synthesizes current knowledge on etiologies, clinical presentations, transplant evaluation, the transformative impact of VADs, and contemporary post-transplant management, aiming to equip clinicians with an updated framework for complex decision-making and optimizing outcomes in this high-risk population.
小儿心肌病(CMP)的发病率约为十万分之一,仍然是一项严峻的临床挑战。这些心肌疾病通常呈进行性发展,常常最终导致终末期心力衰竭,使CMP成为小儿心脏移植的主要指征。移植的关键考虑因素包括特定的CMP病因和亚型,以及既定的列入移植名单标准和应对等待名单复杂性的策略。机械循环支持,尤其是心室辅助装置(VAD)的作用不断演变,改变了过渡策略,并显著提高了移植生存率。当代移植后结果显示持续改善,生存率有所提高,基本长期管理策略也在不断完善。本综述对进展为需要移植的终末期心力衰竭的小儿CMP进行了全面分析。它综合了有关病因、临床表现、移植评估、VAD的变革性影响以及当代移植后管理的现有知识,旨在为临床医生提供一个最新的框架,以便在这一高风险人群中进行复杂的决策并优化治疗结果。