Livingston Sherry I, Durkalski-Mauldin Valerie
Department of Public Health Sciences, Medical University of South Carolina, Charleston, South Carolina.
Gastro Hep Adv. 2022 Apr 29;1(4):538-545. doi: 10.1016/j.gastha.2022.02.026. eCollection 2022.
Acute liver failure (ALF) is a rare but serious disease with challenging clinical decisions, including the possibility of liver transplantation. Although there is interest in predicting who will need a transplant, that outcome is difficult to define as the decision to transplant includes many extraneous factors. The majority of research in this setting focuses on identifying factors that can provide guidance on a patient's likelihood of survival without a liver transplant. The question that arises is whether death and transplant should be combined as a poor outcome or should alternative approaches be used to account for transplant in this setting. Furthermore, does the approach to incorporating transplant information impact the accuracy of predicting survival. We aim to compare alternative analytic methods for the ALF setting to provide guidance to the clinical research community on how to handle transplant when the outcome of interest is survival without a transplant.
Five analysis approaches are compared based on model performance using existing registry data from 2100 ALF patients: logistic regression with transplant as part of the outcome, logistic regression with transplant as a covariate, inverse probability weighting, survival analysis, and multiple imputation.
The various models exhibit comparable model fit with each providing advantages and challenges in implementation.
There are alternative modeling approaches in the ALF setting, leaving researchers with multiple valid options for how to include transplant when examining factors that may influence transplant-free survival.
急性肝衰竭(ALF)是一种罕见但严重的疾病,临床决策具有挑战性,包括肝移植的可能性。尽管人们对预测谁需要进行移植很感兴趣,但由于移植决策包含许多外在因素,因此这一结果很难界定。在这种情况下,大多数研究都集中在识别能够为患者在不进行肝移植的情况下存活可能性提供指导的因素。由此产生的问题是,死亡和移植应被合并视为不良结局,还是应采用其他方法来考虑这种情况下的移植情况。此外,纳入移植信息的方法是否会影响生存预测的准确性。我们旨在比较急性肝衰竭情况下的替代分析方法,为临床研究界提供指导,说明当关注的结局是不进行移植的生存情况时如何处理移植问题。
基于模型性能,使用来自2100例急性肝衰竭患者的现有登记数据,比较五种分析方法:将移植作为结局一部分的逻辑回归、将移植作为协变量的逻辑回归、逆概率加权、生存分析和多重插补。
各种模型表现出可比的模型拟合度,每种方法在实施中都有优点和挑战。
在急性肝衰竭情况下有多种建模方法,这为研究人员在研究可能影响无移植生存的因素时如何纳入移植情况提供了多种有效的选择。