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估计器官移植治疗方案的因果效应。

Estimating the causal effect of treatment regimes for organ transplantation.

作者信息

Boatman Jeffrey A, Vock David M

机构信息

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.

出版信息

Biometrics. 2018 Dec;74(4):1407-1416. doi: 10.1111/biom.12921. Epub 2018 Jul 10.

DOI:10.1111/biom.12921
PMID:29992533
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9119287/
Abstract

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.

摘要

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。

相似文献

1
Estimating the causal effect of treatment regimes for organ transplantation.估计器官移植治疗方案的因果效应。
Biometrics. 2018 Dec;74(4):1407-1416. doi: 10.1111/biom.12921. Epub 2018 Jul 10.
2
Transportability of causal inference under random dynamic treatment regimes for kidney-pancreas transplantation.随机动态治疗方案下肾胰联合移植因果推断的可转移性。
Biometrics. 2023 Dec;79(4):3165-3178. doi: 10.1111/biom.13899. Epub 2023 Jul 10.
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Imputation-based Q-learning for optimizing dynamic treatment regimes with right-censored survival outcome.基于插补的 Q 学习优化右删失生存结局的动态治疗方案。
Biometrics. 2023 Dec;79(4):3676-3689. doi: 10.1111/biom.13872. Epub 2023 May 17.
4
The UNOS OPTN waiting list: 1988-1995.器官共享联合网络(UNOS)等待名单:1988 - 1995年
Clin Transpl. 1995:69-84.
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Transplantation in Canada: review of the last decade from the Canadian Organ Replacement Register.加拿大的移植情况:基于加拿大器官替代登记处对过去十年的回顾。
Clin Transpl. 2003:101-8.
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The UNOS OPTN waiting list and donor registry.器官共享联合网络(UNOS)的器官分配等待名单和捐赠者登记系统。
Clin Transpl. 1997:61-80.
7
Organ donation and transplantation in the UK-the last decade: a report from the UK national transplant registry.英国的器官捐赠与移植:过去十年——来自英国国家移植登记处的报告。
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On logistic regression with right censored data, with or without competing risks, and its use for estimating treatment effects.关于带有右删失数据、有或无竞争风险的逻辑回归及其在估计治疗效果中的应用。
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The UNOS OPTN waiting list, 1988-1998.1988 - 1998年美国器官共享联合网络(UNOS)器官分配等待名单
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The OPTN waiting list, 1988-2003.器官共享联合网络等待名单,1988 - 2003年。
Clin Transpl. 2004:27-40.

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Stat Med. 2022 Sep 20;41(21):4176-4199. doi: 10.1002/sim.9503. Epub 2022 Jul 9.
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Reinforcement Learning in Neurocritical and Neurosurgical Care: Principles and Possible Applications.神经危重症与神经外科学中的强化学习:原则与可能的应用。
Comput Math Methods Med. 2021 Feb 22;2021:6657119. doi: 10.1155/2021/6657119. eCollection 2021.
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A scoping review of studies using observational data to optimise dynamic treatment regimens.使用观察性数据优化动态治疗方案的研究的范围综述。
BMC Med Res Methodol. 2021 Feb 22;21(1):39. doi: 10.1186/s12874-021-01211-2.

本文引用的文献

1
Survival Benefit of Lung Transplantation in the Modern Era of Lung Allocation.现代肺分配时代肺移植的生存获益
Ann Am Thorac Soc. 2017 Feb;14(2):172-181. doi: 10.1513/AnnalsATS.201606-507OC.
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Lung donor selection criteria.肺供体选择标准。
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