Division of Pediatric Cardiology, C.S. Mott Children's Hospital, Ann Arbor, Michigan 48109, USA.
J Heart Lung Transplant. 2009 Dec;28(12):1329-34. doi: 10.1016/j.healun.2009.05.028. Epub 2009 Sep 26.
The course of pediatric hypertrophic cardiomyopathy (HCM) is variable, and the indications for heart transplantation (HTx) are ill defined. This study investigated outcomes and risk factors for adverse outcomes after listing for HTx.
A multicenter, event-driven data registry of patients aged < 18 years listed for HTx between January 1993 and December 2007 was used.
During the study period, 3,147 children were listed for HTx (mean age, 7.6 +/- 0.8 years). Of l,320 with CM at listing, 77 (6%) had HCM (61% boys; 79% white); 59% were United Network of Organ Sharing (UNOS) status I, 30% were receiving inotropes, 27% were ventilated, and 8% required extracorporeal membrane oxygenation. Arrhythmia had occurred in 27%, and 14% had failure to thrive. Within 1 year, 65% underwent HTx. Overall, 25 patients died after listing: 11 (14%) while waiting and 14 of 49 (29%) after HTx. Pre-HTx survival was lower for those listed at age < 1 year (p = 0.0005). Risk factors for death after listing included UNOS status 1 (p = 0.01) and younger age (relative risk, 2.3; p = 0.001). Late (10-year) survival after HTx for HCM patients was 47% vs 63% for non-CM patients within the database.
Children with HCM listed for HTx age < 1 year and UNOS status 1 have the highest mortality awaiting HTx. A more rigorous identification of additional risk factors should be performed to better define timing of listing and which patient sub-group may derive optimal benefit from HTx.
儿科肥厚型心肌病（HCM）的病程变化不定，心脏移植（HTx）的适应证也尚未明确。本研究旨在探讨 HTx 适应证患儿接受 HTx 后发生不良结局的预后因素。
采用多中心、事件驱动型数据登记系统，对 1993 年 1 月至 2007 年 12 月期间年龄＜18 岁的 HTx 适应证患儿进行登记。
研究期间，共有 3147 例患儿接受 HTx 适应证登记（平均年龄 7.6±0.8 岁）。1320 例心肌病患儿中，77 例（6%）为 HCM（61%为男性；79%为白人）；59%为美国器官共享联合网络（UNOS）Ⅰ级，30%接受正性肌力药物治疗，27%需要机械通气，8%需要体外膜肺氧合。27%患儿出现心律失常，14%存在生长发育迟缓。1 年内，65%患儿接受 HTx。总体而言，25 例患儿在登记后死亡：11 例（14%）在等待期间死亡，14 例（29%）在 HTx 后死亡。1 岁以下患儿的 HTx 前生存率较低（p=0.0005）。登记后死亡的危险因素包括 UNOS Ⅰ级（p=0.01）和年龄较小（相对危险度 2.3；p=0.001）。HTx 后 10 年时，HCM 患儿的总体生存率为 47%，数据库中非 CM 患儿的生存率为 63%。
＜1 岁且 UNOS Ⅰ级的 HCM 患儿接受 HTx 等待期间死亡率最高。需要进一步明确其他风险因素，以便更好地确定登记时间，并明确哪些亚组患者可能从 HTx 中获益最大。
