Boatman Jeffrey A, Vock David M
Division of Biostatistics, School of Public Health, University of Minnesota, A460 Mayo Building, MMC 303, 420 Delaware St. SE, Minneapolis, Minnesota 55455, U.S.A.
Biometrics. 2018 Dec;74(4):1407-1416. doi: 10.1111/biom.12921. Epub 2018 Jul 10.
Patients awaiting cadaveric organ transplantation face a difficult decision if offered a low-quality organ: accept the organ or remain on the waiting list and hope a better organ is offered in the future. A dynamic treatment regime (DTR) for transplantation is a rule that determines whether a patient should decline an offered organ. Existing methods can estimate the effect of DTRs on survival outcomes, but these were developed for applications where treatment is abundantly available. For transplantation, organ availability is limited, and existing methods can only estimate the effect of a DTR assuming a single patient follows the DTR. We show for transplantation that the effect of a DTR depends on whether other patients follow the DTR. To estimate the anticipated survival if the entire population awaiting transplantation were to adopt a DTR, we develop a novel inverse probability weighted estimator (IPCW) which re-weights patients based on the probability of following their transplant history in the counterfactual world in which all patients follow the DTR of interest. We estimate this counterfactual probability using hot deck imputation to fill in data that is not observed for patients who are artificially censored by IPCW once they no longer follow the DTR of interest. We show via simulation that our proposed method has good finite-sample properties, and we apply our method to a lung transplantation observational registry.
等待尸体器官移植的患者如果获得一个质量不佳的器官,就会面临艰难抉择:接受该器官还是继续留在等待名单上,期望未来能获得一个更好的器官。一种用于移植的动态治疗方案(DTR)是一种确定患者是否应拒绝所提供器官的规则。现有方法可以估计DTR对生存结果的影响,但这些方法是为治疗资源丰富的应用场景开发的。对于移植而言,器官供应有限,并且现有方法只能在假设单个患者遵循DTR的情况下估计DTR的效果。我们针对移植表明,DTR的效果取决于其他患者是否遵循该DTR。为了估计如果所有等待移植的患者都采用一种DTR时的预期生存率,我们开发了一种新颖的逆概率加权估计器(IPCW),该估计器根据在所有患者都遵循感兴趣的DTR的反事实世界中遵循其移植历史的概率对患者进行重新加权。我们使用热卡插补来估计这种反事实概率,以填补那些一旦不再遵循感兴趣的DTR就被IPCW人为删失的患者未观察到的数据。我们通过模拟表明,我们提出的方法具有良好的有限样本性质,并将我们的方法应用于一个肺移植观察性登记处。