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基于模型森林的观察性数据异质处理效应估计。

Heterogeneous treatment effect estimation for observational data using model-based forests.

机构信息

Institut für Statistik, Ludwig-Maximilians-Universität München, Munich, Germany.

Munich Center for Machine Learning (MCML), Germany.

出版信息

Stat Methods Med Res. 2024 Mar;33(3):392-413. doi: 10.1177/09622802231224628. Epub 2024 Feb 8.

DOI:10.1177/09622802231224628
PMID:38332489
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10981193/
Abstract

The estimation of heterogeneous treatment effects has attracted considerable interest in many disciplines, most prominently in medicine and economics. Contemporary research has so far primarily focused on continuous and binary responses where heterogeneous treatment effects are traditionally estimated by a linear model, which allows the estimation of constant or heterogeneous effects even under certain model misspecifications. More complex models for survival, count, or ordinal outcomes require stricter assumptions to reliably estimate the treatment effect. Most importantly, the noncollapsibility issue necessitates the joint estimation of treatment and prognostic effects. Model-based forests allow simultaneous estimation of covariate-dependent treatment and prognostic effects, but only for randomized trials. In this paper, we propose modifications to model-based forests to address the confounding issue in observational data. In particular, we evaluate an orthogonalization strategy originally proposed by Robinson (1988, Econometrica) in the context of model-based forests targeting heterogeneous treatment effect estimation in generalized linear models and transformation models. We found that this strategy reduces confounding effects in a simulated study with various outcome distributions. We demonstrate the practical aspects of heterogeneous treatment effect estimation for survival and ordinal outcomes by an assessment of the potentially heterogeneous effect of Riluzole on the progress of Amyotrophic Lateral Sclerosis.

摘要

在许多学科中,特别是在医学和经济学中,异质处理效应的估计引起了相当大的兴趣。目前的研究主要集中在连续和二项反应上,传统上通过线性模型来估计异质处理效应,该模型允许在某些模型失拟的情况下估计常数或异质效应。对于生存、计数或有序结果的更复杂模型,需要更严格的假设才能可靠地估计处理效果。最重要的是,非 collapsible 问题需要联合估计治疗效果和预后效果。基于模型的森林允许同时估计依赖协变量的治疗和预后效果,但仅适用于随机试验。在本文中,我们提出了对基于模型的森林的修改,以解决观察数据中的混杂问题。具体来说,我们评估了 Robinson(1988,Econometrica)提出的一种正交化策略,该策略针对广义线性模型和转换模型中的异质处理效应估计,目标是基于模型的森林。我们发现,这种策略在具有各种结果分布的模拟研究中减少了混杂效应。我们通过评估利鲁唑对肌萎缩侧索硬化症进展的潜在异质效应,演示了生存和有序结果的异质处理效应估计的实际方面。

相似文献

1
Heterogeneous treatment effect estimation for observational data using model-based forests.基于模型森林的观察性数据异质处理效应估计。
Stat Methods Med Res. 2024 Mar;33(3):392-413. doi: 10.1177/09622802231224628. Epub 2024 Feb 8.
2
Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND).利鲁唑用于治疗肌萎缩侧索硬化症(ALS)/运动神经元病(MND)。
Amyotroph Lateral Scler Other Motor Neuron Disord. 2003 Sep;4(3):191-206.
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Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND).利鲁唑用于治疗肌萎缩侧索硬化症(ALS)/运动神经元病(MND)。
Cochrane Database Syst Rev. 2002(2):CD001447. doi: 10.1002/14651858.CD001447.
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Folic acid supplementation and malaria susceptibility and severity among people taking antifolate antimalarial drugs in endemic areas.在流行地区,服用抗叶酸抗疟药物的人群中,叶酸补充剂与疟疾易感性和严重程度的关系。
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Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND).利鲁唑用于治疗肌萎缩侧索硬化症(ALS)/运动神经元病(MND)。
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Comparing methods for estimation of heterogeneous treatment effects using observational data from health care databases.利用医疗保健数据库中的观察数据比较估计异质治疗效果的方法。
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Survival forests under test: Impact of the proportional hazards assumption on prognostic and predictive forests for amyotrophic lateral sclerosis survival.测试中的生存森林:比例风险假设对肌萎缩侧索硬化症生存的预后和预测森林的影响。
Stat Methods Med Res. 2020 May;29(5):1403-1419. doi: 10.1177/0962280219862586. Epub 2019 Jul 15.
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Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND).利鲁唑用于治疗肌萎缩侧索硬化症(ALS)/运动神经元病(MND)。
Cochrane Database Syst Rev. 2012 Mar 14;2012(3):CD001447. doi: 10.1002/14651858.CD001447.pub3.

本文引用的文献

1
Comparing algorithms for characterizing treatment effect heterogeneity in randomized trials.比较随机试验中刻画处理效应异质性的算法。
Biom J. 2024 Jan;66(1):e2100337. doi: 10.1002/bimj.202100337. Epub 2022 Nov 27.
2
Core concepts in pharmacoepidemiology: Violations of the positivity assumption in the causal analysis of observational data: Consequences and statistical approaches.药物流行病学的核心概念:观察性数据分析中因果关系分析中阳性假设的违背:后果和统计方法。
Pharmacoepidemiol Drug Saf. 2021 Nov;30(11):1471-1485. doi: 10.1002/pds.5338. Epub 2021 Aug 24.
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Estimating heterogeneous survival treatment effect in observational data using machine learning.
利用机器学习估计观察性数据中异质生存治疗效果。
Stat Med. 2021 Sep 20;40(21):4691-4713. doi: 10.1002/sim.9090. Epub 2021 Jun 10.
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Making apples from oranges: Comparing noncollapsible effect estimators and their standard errors after adjustment for different covariate sets.将橘子变成苹果:比较在调整不同协变量集后不可折叠的效应估计量及其标准误差。
Biom J. 2021 Mar;63(3):528-557. doi: 10.1002/bimj.201900297. Epub 2020 Dec 14.
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Model-based random forests for ordinal regression.用于有序回归的基于模型的随机森林
Int J Biostat. 2020 Aug 7. doi: 10.1515/ijb-2019-0063.
6
Real-world evidence of riluzole effectiveness in treating amyotrophic lateral sclerosis.瑞鲁唑治疗肌萎缩侧索硬化症的真实世界证据。
Amyotroph Lateral Scler Frontotemporal Degener. 2020 Nov;21(7-8):509-518. doi: 10.1080/21678421.2020.1771734. Epub 2020 Jun 23.
7
Estimation of causal effects of multiple treatments in observational studies with a binary outcome.二元结局观察性研究中多种治疗因果效应的估计。
Stat Methods Med Res. 2020 Nov;29(11):3218-3234. doi: 10.1177/0962280220921909. Epub 2020 May 25.
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Non-parametric individual treatment effect estimation for survival data with random forests.基于随机森林的生存数据的非参数个体治疗效果估计。
Bioinformatics. 2020 Jan 15;36(2):629-636. doi: 10.1093/bioinformatics/btz602.
9
Survival forests under test: Impact of the proportional hazards assumption on prognostic and predictive forests for amyotrophic lateral sclerosis survival.测试中的生存森林:比例风险假设对肌萎缩侧索硬化症生存的预后和预测森林的影响。
Stat Methods Med Res. 2020 May;29(5):1403-1419. doi: 10.1177/0962280219862586. Epub 2019 Jul 15.
10
A comparison of subgroup identification methods in clinical drug development: Simulation study and regulatory considerations.临床药物开发中亚组识别方法的比较:模拟研究与监管考量
Pharm Stat. 2019 Oct;18(5):600-626. doi: 10.1002/pst.1951. Epub 2019 Jul 3.