Department of Pediatric Cardiology, Freeman Hospital, Newcastle upon Tyne, United Kingdom.
J Heart Lung Transplant. 2009 Dec;28(12):1322-8. doi: 10.1016/j.healun.2009.05.027. Epub 2009 Sep 26.
The course of dilated cardiomyopathy (DCM) leading to heart failure in children varies; survival with conventional treatment is 64% at 5 years. Heart transplantation (HTx) enables improved survival; however, outcomes from listing for transplant are not well described. This study reports survival of patients with DCM from listing with the availability of mechanical bridge to transplant.
Patients with a primary diagnosis of DCM (n = 1,098) were identified from a multi-institutional, prospective, registry of patients aged < 18 years listed for HTx from January 1, 1993, to December 31, 2006.
Characteristics of DCM patients at listing included a mean age of 7.3 years; 51% male, 64% white ethnicity, 77% United Network for Organ Sharing status I, 66% on inotropic support, 28% mechanically ventilated, and 15% on mechanical support. Waitlist mortality was 11%, and 75% underwent HTx at 2 years after listing. Overall 10-year survival after listing was 72%, with higher risk of death associated with arrhythmias, mechanical ventilation, and extracorporeal membrane oxygenation (ECMO) support, but not ventricular assist device (VAD) support. Survival at 10 years post-HTx was 72%, with a higher risk of death associated with black race, older age, mechanical ventilation, longer ischemic time, and earlier era of transplant.
Transplantation for DCM in the pediatric population offers enhanced survival compared with the natural history. Overall waitlist mortality for DCM is low, with the exception of patients on ECMO, mechanically ventilated, or with arrhythmias. DCM patients fared well after transplant, making HTx a key therapeutic intervention.
导致儿童心力衰竭的扩张型心肌病（DCM）病程各异；采用常规治疗，5 年生存率为 64%。心脏移植（HTx）可提高生存率；然而，接受移植的患者预后情况尚不清楚。本研究报告了从列入移植名单至获得机械桥接进行移植的 DCM 患者的生存率。
从 1993 年 1 月 1 日至 2006 年 12 月 31 日，一项多机构、前瞻性、接受 HTx 的<18 岁患者注册研究中，确定了患有 DCM 的患者（n=1098）。
列入名单的 DCM 患者特征包括平均年龄为 7.3 岁；51%为男性，64%为白人，77%为 United Network for Organ Sharing 状态 I，66%接受正性肌力支持，28%机械通气，15%接受机械支持。等待名单死亡率为 11%，75%在列入名单后 2 年内接受 HTx。列入名单后 10 年总体生存率为 72%，心律失常、机械通气和体外膜肺氧合（ECMO）支持与较高的死亡风险相关，但心室辅助装置（VAD）支持则不然。HTx 后 10 年生存率为 72%，黑人种族、年龄较大、机械通气、缺血时间较长和移植早期的患者死亡风险较高。
与自然病史相比，儿科人群接受 DCM 移植可提高生存率。除 ECMO、机械通气或心律失常患者外，DCM 患者的总体等待名单死亡率较低。DCM 患者在移植后预后良好，使 HTx 成为关键的治疗干预措施。
