Department of Pediatrics (Cardiology), Stanford University School of Medicine, Palo Alto, California, USA.
Department of Economics, Stanford University, Palo Alto, California, USA.
J Am Coll Cardiol. 2024 Aug 13;84(7):620-632. doi: 10.1016/j.jacc.2024.05.049.
In 2016, the United Network for Organ Sharing revised its pediatric heart transplant (HT) allocation policy.
This study sought to determine whether the 2016 revisions are associated with reduced waitlist mortality and capture patient-specific risks.
Children listed for HT from 1999 to 2023 were identified using Organ Procurement and Transplantation Network data and grouped into 3 eras (era 1: 1999-2006; era 2: 2006-2016; era 3: 2016-2023) based on when the United Network for Organ Sharing implemented allocation changes. Fine-Gray competing risks modeling was used to identify factors associated with death or delisting for deterioration. Fixed-effects analysis was used to determine whether allocation changes were associated with mortality.
Waitlist mortality declined 8 percentage points (PP) across eras (21%, 17%, and 13%, respectively; P < 0.01). At listing, era 3 children were less sick than era 1 children, with 6 PP less ECMO use (P < 0.01), 11 PP less ventilator use (P < 0.01), and 1 PP less dialysis use (P < 0.01). Ventricular assist device (VAD) use was 13 PP higher, and VAD mortality decreased 9 PP (P < 0.01). Non-White mortality declined 10 PP (P < 0.01). ABO-incompatible listings increased 27 PP, and blood group O infant mortality decreased 13 PP (P < 0.01). In multivariable analyses, the 2016 revisions were not associated with lower waitlist mortality, whereas VAD use (in era 3), ABO-incompatible transplant, improved patient selection, and narrowing racial disparities were. Match-run analyses demonstrated poor correlation between individual waitlist mortality risk and the match-run order.
The 2016 allocation revisions were not independently associated with the decline in pediatric HT waitlist mortality. The 3-tier classification system fails to adequately capture patient-specific risks. A more flexible allocation system that accurately reflects patient-specific risks and considers transplant benefit is urgently needed.
2016 年,美国器官共享网络修订了儿科心脏移植(HT)分配政策。
本研究旨在确定 2016 年的修订是否与降低候补名单死亡率和捕捉患者特定风险有关。
使用器官获取与移植网络数据确定 1999 年至 2023 年接受 HT 名单的儿童,并根据美国器官共享网络实施分配变更的时间将其分为 3 个时期(时期 1:1999-2006 年;时期 2:2006-2016 年;时期 3:2016-2023 年)。采用 Fine-Gray 竞争风险模型确定与死亡或降级除名相关的因素。采用固定效应分析确定分配变更是否与死亡率相关。
各时期候补名单死亡率均下降 8 个百分点(分别为 21%、17%和 13%;P<0.01)。在名单上时,第 3 时期的患儿比第 1 时期的患儿病情较轻,体外膜肺氧合(ECMO)使用率低 6 个百分点(P<0.01),呼吸机使用率低 11 个百分点(P<0.01),透析使用率低 1 个百分点(P<0.01)。心室辅助装置(VAD)使用率高 13 个百分点,VAD 死亡率下降 9 个百分点(P<0.01)。非白人死亡率下降 10 个百分点(P<0.01)。ABO 不相容名单增加 27 个百分点,婴儿血型 O 死亡率下降 13 个百分点(P<0.01)。多变量分析显示,2016 年修订与候补名单死亡率降低无关,而 VAD 使用(第 3 时期)、ABO 不相容移植、改善患者选择以及缩小种族差异有关。匹配运行分析表明,个别候补名单死亡率风险与匹配运行顺序之间相关性较差。
2016 年分配修订与儿科 HT 候补名单死亡率的下降无关。三级分类系统不能充分捕捉患者特定的风险。迫切需要建立一种更灵活的分配系统,该系统能够准确反映患者特定的风险并考虑移植获益。