University of Texas Southwestern, Department of Pediatrics, Division of Cardiology, Dallas (R.J.B.).
Children's Medical Center of Dallas, TX (L.T.).
Circulation. 2024 Jul 30;150(5):362-373. doi: 10.1161/CIRCULATIONAHA.123.068189. Epub 2024 Jun 28.
Waitlist mortality (WM) remains elevated in pediatric heart transplantation. Allocation policy is a potential tool to help improve WM. This study aims to identify patients at highest risk for WM to potentially inform future allocation policy changes.
The Pediatric Heart Transplant Society database was queried for patients <18 years of age indicated for heart transplantation between January 1, 2010 to December 31, 2021. was defined as death while awaiting transplant or removal from the waitlist due to clinical deterioration. Because WM is low after the first year, analysis was limited to the first 12 months on the heart transplant list. Kaplan-Meier analysis and log-rank testing was conducted to compare unadjusted survival between groups. Cox proportional hazard models were created to determine risk factors for WM. Subgroup analysis was performed for status 1A patients based on body surface area (BSA) at time of listing, cardiac diagnosis, and presence of mechanical circulatory support.
In total 5974 children met study criteria of which 3928 were status 1A, 1012 were status 1B, 963 were listed status 2, and 65 were listed status 7. Because of the significant burden of WM experienced by 1A patients, further analysis was performed in only patients indicated as 1A. Within that group of patients, those with smaller size and lower eGFR had higher WM, whereas those patients without congenital heart disease or support from a ventricular assist device (VAD) at time of listing had decreased WM. In the smallest size cohort, cardiac diagnoses other than dilated cardiomyopathy were risk factors for WM. Previous cardiac surgery was a risk factor in the 0.3 to 0.7 m and >0.7 m BSA groups. VAD support was associated with lower WM other than in the single ventricle cohort, where VAD was associated with higher WM. Extracorporeal membrane oxygenation and mechanical ventilation were associated with increased risk of WM in all cohorts.
There is significant variability in WM among status-1A patients. Potential refinements to current allocation system should factor in the increased WM risk we identified in patients supported by extracorporeal membrane oxygenation or mechanical ventilation, single ventricle congenital heart disease on VAD support and small children with congenital heart disease, restrictive cardiomyopathy, or hypertrophic cardiomyopathy.
儿科心脏移植患者的候补者死亡率（WM）仍然居高不下。分配政策是帮助改善 WM 的潜在工具。本研究旨在确定 WM 风险最高的患者，以便为未来的分配政策变化提供信息。
查询了 2010 年 1 月 1 日至 2021 年 12 月 31 日期间接受心脏移植的年龄<18 岁的患者的儿科心脏移植协会数据库。WM 定义为在等待移植或因临床恶化而从候补名单中除名期间死亡。由于 WM 在第一年之后很低，因此分析仅限于候补名单上的前 12 个月。进行 Kaplan-Meier 分析和对数秩检验以比较组间未调整的生存率。创建 Cox 比例风险模型以确定 WM 的危险因素。根据列表时的体表面积 (BSA)、心脏诊断和机械循环支持的存在，对 1A 状态患者进行亚组分析。
共有 5974 名儿童符合研究标准，其中 3928 名是 1A 状态，1012 名是 1B 状态，963 名是 2 状态，65 名是 7 状态。由于 1A 患者 WM 负担过重，因此仅在被列为 1A 的患者中进行了进一步分析。在该组患者中，体型较小和 eGFR 较低的患者 WM 较高，而在列表中没有先天性心脏病或心室辅助装置（VAD）支持的患者 WM 较低。在最小体型队列中，除扩张型心肌病以外的心脏诊断是 WM 的危险因素。心脏手术是 0.3 至 0.7 m 和>0.7 m BSA 组的危险因素。VAD 支持与除单心室队列外的 WM 较低有关，而在单心室队列中，VAD 与 WM 较高有关。体外膜氧合和机械通气与所有队列中的 WM 风险增加有关。
1A 状态患者之间的 WM 存在显著差异。当前分配系统的潜在改进应考虑到我们在接受体外膜氧合或机械通气支持、单心室先天性心脏病患者接受 VAD 支持以及患有先天性心脏病、限制型心肌病或肥厚型心肌病的小型儿童中发现的 WM 风险增加。
