Robust Causal Inference for EHR-based Studies of Point Exposures with Missingness in Eligibility Criteria

Fuente: arXiv
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Main Authors: Benz, Luke, Mukherjee, Rajarshi, Wang, Rui, Arterburn, David, Fischer, Heidi, Lee, Catherine, Shortreed, Susan M., Haneuse, Sebastien, Levis, Alexander W.
Format: Preprint
Published: 2025
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_version_ 1866913095915405312
author Benz, Luke
Mukherjee, Rajarshi
Wang, Rui
Arterburn, David
Fischer, Heidi
Lee, Catherine
Shortreed, Susan M.
Haneuse, Sebastien
Levis, Alexander W.
author_facet Benz, Luke
Mukherjee, Rajarshi
Wang, Rui
Arterburn, David
Fischer, Heidi
Lee, Catherine
Shortreed, Susan M.
Haneuse, Sebastien
Levis, Alexander W.
contents Missingness in variables that define study eligibility criteria is a seldom addressed challenge in electronic health record (EHR)-based settings. It is typically the case that patients with incomplete eligibility information are excluded from analysis without consideration of (implicit) assumptions that are being made, leaving study conclusions subject to potential selection bias. In an effort to ascertain eligibility for more patients, researchers may look back further in time prior to study baseline, and in using outdated values of eligibility-defining covariates may inappropriately be including individuals who, unbeknownst to the researcher, fail to meet eligibility at baseline. To the best of our knowledge, however, very little work has been done to mitigate these concerns. We propose a robust and efficient estimator of the causal average treatment effect on the treated, defined in the study eligible population, in cohort studies where eligibility-defining covariates are missing at random. The approach facilitates the use of flexible machine-learning strategies for component nuisance functions while maintaining appropriate convergence rates for valid asymptotic inference. This method is directly motivated by, and applied throughout to EHR data from Kaiser Permanente to analyze differences between two common bariatric surgical interventions for long-term weight and glycemic outcomes among a cohort of severely obese patients with type II diabetes mellitus.
format Preprint
id arxiv_https___arxiv_org_abs_2504_16230
institution arXiv
publishDate 2025
record_format arxiv
spellingShingle Robust Causal Inference for EHR-based Studies of Point Exposures with Missingness in Eligibility Criteria
Benz, Luke
Mukherjee, Rajarshi
Wang, Rui
Arterburn, David
Fischer, Heidi
Lee, Catherine
Shortreed, Susan M.
Haneuse, Sebastien
Levis, Alexander W.
Methodology
Applications
Missingness in variables that define study eligibility criteria is a seldom addressed challenge in electronic health record (EHR)-based settings. It is typically the case that patients with incomplete eligibility information are excluded from analysis without consideration of (implicit) assumptions that are being made, leaving study conclusions subject to potential selection bias. In an effort to ascertain eligibility for more patients, researchers may look back further in time prior to study baseline, and in using outdated values of eligibility-defining covariates may inappropriately be including individuals who, unbeknownst to the researcher, fail to meet eligibility at baseline. To the best of our knowledge, however, very little work has been done to mitigate these concerns. We propose a robust and efficient estimator of the causal average treatment effect on the treated, defined in the study eligible population, in cohort studies where eligibility-defining covariates are missing at random. The approach facilitates the use of flexible machine-learning strategies for component nuisance functions while maintaining appropriate convergence rates for valid asymptotic inference. This method is directly motivated by, and applied throughout to EHR data from Kaiser Permanente to analyze differences between two common bariatric surgical interventions for long-term weight and glycemic outcomes among a cohort of severely obese patients with type II diabetes mellitus.
title Robust Causal Inference for EHR-based Studies of Point Exposures with Missingness in Eligibility Criteria
topic Methodology
Applications
url https://arxiv.org/abs/2504.16230