Department of Student Affairs, Baylor College of Medicine, Houston, USA.
Division of Abdominal Transplantation, Michael E DeBakey Department of General Surgery, Baylor College of Medicine, Houston, USA.
Can J Gastroenterol Hepatol. 2023 Mar 2;2023:2859384. doi: 10.1155/2023/2859384. eCollection 2023.
The impact of indication for pediatric liver transplantation on waitlist and post-transplant mortality outcomes is well known, but the impact on intent-to-treat outcomes has not been investigated. Intent-to-treat survival analysis is important in this study because it is more comprehensive, combining the transplant outcomes of waitlist mortality, post-transplant mortality, and transplant rate into a single metric to elucidate any disparities in outcomes based on indication.
Cox regression was used to analyze factors impacting survival in 8,002 children listed for liver transplant in the UNOS database between 2006 and 2016. The Kaplan-Meier method and log-rank test were used to assess differences in waitlist, post-transplant, and intent-to-treat mortality among the top 5 indications of biliary atresia, acute hepatic necrosis, metabolic disorders, hepatoblastoma, and autoimmune cirrhosis.
When compared to the reference group of biliary atresia, multivariate analyses showed that every indication was associated with inferior intent-to-treat outcomes except for metabolic disorders. Hepatoblastoma (hazard ratio (HR): 3.73), autoimmune cirrhosis (HR: 1.86), and AHN (HR: 1.77) were associated with significantly increased intent-to-treat mortality. Hepatoblastoma was also associated with increased post-transplant mortality (HR: 3.77) and was the only indication significantly associated with increased waitlist mortality (HR: 6.43).
Significant disparity exists across all indications with respect to an increased intent-to-treat mortality, along with an increased post-transplant and waitlist mortality, when compared to the biliary atresia reference group. If further studies validate these findings, a reexamination of the equitable distribution of allografts for transplant may be warranted as well as a focus on disparities in survival after transplant.
儿科肝移植的适应证对等待名单和移植后死亡率结果的影响是众所周知的，但对意向治疗结果的影响尚未得到研究。在这项研究中，意向治疗生存分析很重要，因为它更全面，将等待名单死亡率、移植后死亡率和移植率的移植结果结合到一个单一的指标中，根据适应证阐明任何结果差异。
使用 Cox 回归分析 2006 年至 2016 年 UNOS 数据库中 8002 例儿童肝移植的生存因素。使用 Kaplan-Meier 方法和对数秩检验评估胆道闭锁、急性肝坏死、代谢紊乱、肝母细胞瘤和自身免疫性肝硬化这 5 种主要适应证的等待名单、移植后和意向治疗死亡率之间的差异。
与胆道闭锁的参考组相比，多变量分析显示，除代谢紊乱外，每一种适应证都与较差的意向治疗结果相关。肝母细胞瘤（风险比（HR）：3.73）、自身免疫性肝硬化（HR：1.86）和 AHN（HR：1.77）与意向治疗死亡率显著增加相关。肝母细胞瘤还与移植后死亡率增加（HR：3.77）相关，是唯一与等待名单死亡率增加显著相关的适应证（HR：6.43）。
与胆道闭锁参考组相比，所有适应证在意向治疗死亡率增加的情况下，以及移植后和等待名单死亡率增加的情况下，都存在显著的差异。如果进一步的研究验证了这些发现，可能需要重新考虑移植同种异体移植物的公平分配，以及关注移植后生存的差异。
