Department of Pediatrics, Washington University in St. Louis, St. Louis Children's Hospital, St. Louis, MO, USA.
Division of Biostatistics, Washington University in St. Louis, St. Louis, MO, USA.
Pediatr Transplant. 2021 May;25(3):e13932. doi: 10.1111/petr.13932. Epub 2020 Nov 24.
Young children listed for liver transplant have high waitlist mortality (WL), which is not fully predicted by the PELD score. SRTR database was queried for children < 2 years listed for initial LT during 2002-17 (n = 4973). Subjects were divided into three outcome groups: bad (death or removal for too sick to transplant), good (spontaneous improvement), and transplant. Demographic, clinical, listing history, and laboratory variables at the time of listing (baseline variables), and changes in variables between listing and prior to outcome (trajectory variables) were analyzed using random forest (RF) analysis. 81.5% candidates underwent LT, and 12.3% had bad outcome. RF model including both baseline and trajectory variables improved prediction compared to model using baseline variables alone. RF analyses identified change in serum creatinine and listing status as the most predictive variables. 80% of subjects listed with a PELD score at time of listing and outcome underwent LT, while ~70% of subjects in both bad and good outcome groups were listed with either Status 1 (A or B) prior to an outcome, regardless of initial listing status. Increase in creatinine on LT waitlist was predictive of bad outcome. Longer time spent on WL was predictive of good outcome. Subjects with biliary atresia, liver tumors, and metabolic disease had LT rate >85%, while >20% of subjects with acute liver failure had a bad outcome. Change in creatinine, listing status, need for RRT, time spent on LT waitlist, and diagnoses were the most predictive variables.
2 岁以下儿童肝移植等待名单死亡率较高(WL),PELD 评分并不能完全预测。本研究通过查询 SRTR 数据库,获取了 2002 年至 2017 年期间首次 LT 列入名单的<2 岁儿童(n=4973)。将受试者分为三组:预后不良(死亡或因病情太重而无法移植)、预后良好(自发改善)和移植。在列入名单时(基线变量)和在结果发生之前(轨迹变量)分析了人口统计学、临床、列入名单历史和实验室变量,以及变量的变化,并使用随机森林(RF)分析。81.5%的候选者接受了 LT,12.3%的患者预后不良。与仅使用基线变量的模型相比,包含基线和轨迹变量的 RF 模型改善了预测。RF 分析确定血清肌酐和列入名单状态的变化是最具预测性的变量。80%的患者在列入名单时和结果时的 PELD 评分进行 LT,而不良和良好结果组中约 70%的患者在发生结果之前都处于状态 1(A 或 B),无论初始列入名单状态如何。LT 等待名单上的肌酐增加是预后不良的预测指标。WL 上花费的时间较长是预后良好的预测指标。胆道闭锁、肝脏肿瘤和代谢疾病患者的 LT 率>85%,而>20%的急性肝衰竭患者预后不良。肌酐变化、列入名单状态、需要 RRT、LT 等待名单上花费的时间和诊断是最具预测性的变量。