Hazard-Based Targeted Maximum Likelihood Estimation for Survival in Resampling Designs
Fuente:
arXiv
Saved in:
| Main Authors: | , , , |
|---|---|
| Format: | Preprint |
| Published: |
2025
|
| Subjects: | |
| Online Access: | |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1866909913360367616 |
|---|---|
| author | Landsiedel, Kirsten E. Phillips, Rachael V. Petersen, Maya L. van der Laan, Mark J. |
| author_facet | Landsiedel, Kirsten E. Phillips, Rachael V. Petersen, Maya L. van der Laan, Mark J. |
| contents | Survival is a key metric for evaluating standards of care for people living with HIV. In resource-limited settings, high rates of loss to follow-up (LTFU) often result in underestimation of mortality when only observed deaths are considered. Resampling, which tracks a subset of LTFU patients to ascertain their outcomes, mitigates bias and improves survival estimates. However, common estimators for survival in resampling designs, such as weighted Kaplan-Meier (KM), fail to leverage covariate information collected during repeated clinic visits, even though this information is highly predictive of survival. We propose a Targeted Maximum Likelihood Estimator (TMLE) for survival in resampling designs, which addresses these limitations by leveraging baseline and longitudinal covariates to achieve greater efficiency. Our TMLE is a plug-in estimator and is robust to misspecification of the initial model for the conditional hazard of death, guaranteeing consistency of our estimator due to known resampling probabilities. We present: (1) a fully efficient TMLE for data from resampling studies with fixed follow-up time for all participants and (2) an inverse probability of censoring weighted (IPCW) TMLE that accounts for varied follow-up times by stratifying on patients with sufficient follow-up to evaluate survival. This IPCW-TMLE can be made highly efficient through nonparametric or targeted estimation of the follow-up censoring mechanism. In simulations, our TMLE reduced variance by up to 55% compared with the commonly used weighted KM estimator while preserving nominal confidence interval coverage. These findings demonstrate the potential of our TMLE to improve survival estimation in resampling designs, offering a robust and resource-efficient framework for HIV research. Keywords: Resampling designs, Survival analysis, Targeted Maximum Likelihood Estimation, Inverse probability weighting |
| format | Preprint |
| id |
arxiv_https___arxiv_org_abs_2511_15045 |
| institution | arXiv |
| publishDate | 2025 |
| record_format | arxiv |
| spellingShingle | Hazard-Based Targeted Maximum Likelihood Estimation for Survival in Resampling Designs Landsiedel, Kirsten E. Phillips, Rachael V. Petersen, Maya L. van der Laan, Mark J. Methodology Survival is a key metric for evaluating standards of care for people living with HIV. In resource-limited settings, high rates of loss to follow-up (LTFU) often result in underestimation of mortality when only observed deaths are considered. Resampling, which tracks a subset of LTFU patients to ascertain their outcomes, mitigates bias and improves survival estimates. However, common estimators for survival in resampling designs, such as weighted Kaplan-Meier (KM), fail to leverage covariate information collected during repeated clinic visits, even though this information is highly predictive of survival. We propose a Targeted Maximum Likelihood Estimator (TMLE) for survival in resampling designs, which addresses these limitations by leveraging baseline and longitudinal covariates to achieve greater efficiency. Our TMLE is a plug-in estimator and is robust to misspecification of the initial model for the conditional hazard of death, guaranteeing consistency of our estimator due to known resampling probabilities. We present: (1) a fully efficient TMLE for data from resampling studies with fixed follow-up time for all participants and (2) an inverse probability of censoring weighted (IPCW) TMLE that accounts for varied follow-up times by stratifying on patients with sufficient follow-up to evaluate survival. This IPCW-TMLE can be made highly efficient through nonparametric or targeted estimation of the follow-up censoring mechanism. In simulations, our TMLE reduced variance by up to 55% compared with the commonly used weighted KM estimator while preserving nominal confidence interval coverage. These findings demonstrate the potential of our TMLE to improve survival estimation in resampling designs, offering a robust and resource-efficient framework for HIV research. Keywords: Resampling designs, Survival analysis, Targeted Maximum Likelihood Estimation, Inverse probability weighting |
| title | Hazard-Based Targeted Maximum Likelihood Estimation for Survival in Resampling Designs |
| topic | Methodology |
| url | https://arxiv.org/abs/2511.15045 |