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A Bayesian Approach for Estimating the Survivor Average Causal Effect When Outcomes Are Truncated by Death in
American Journal of Epidemiology
|February 17, 2023
Summary
This study introduces a new Bayesian method to estimate treatment effects in complex studies, addressing missing data and clustering. The approach provides a clear causal interpretation for the survivor average causal effect (SACE) in specific patient groups.
Area of Science:
- Statistics
- Biostatistics
- Epidemiology
Background:
- Multicenter studies and cluster-randomized trials (CRTs) often face challenges with clustered data and outcomes missing not at random (MNAR).
- Traditional statistical methods struggle with informative truncation and clustering, particularly in vulnerable populations like the elderly or seriously ill.
- Existing causal estimands may be suboptimal for addressing these combined complexities in statistical inference.
Purpose of the Study:
- To develop a Bayesian estimator for the survivor average causal effect (SACE) in clustered/hierarchical data settings.
- To jointly identify the always-survivor principal stratum and estimate the average treatment effect among this group.
- To provide a statistically robust method for handling nonignorable missing outcomes in multicenter and cluster-randomized trials.
Main Methods:
- Utilized principal stratification to define and identify the always-survivor principal stratum.
- Developed a Bayesian estimator to jointly estimate the SACE in a clustered/hierarchical data setting.
- Conducted simulation studies to assess the bias and coverage of the proposed method.
Main Results:
- Simulation studies demonstrated low bias and good coverage for the Bayesian estimator.
- Reanalysis of a motivating cluster-randomized trial showed that the SACE estimate differed in magnitude from complete-case analysis but both were small.
- The SACE estimate provided a clear and rigorous causal interpretation for a specific subset of participants.
Conclusions:
- The developed Bayesian method effectively addresses informative truncation and clustering in statistical inference.
- The survivor average causal effect (SACE) offers valuable insights into treatment effects among survivors in complex study designs.
- Practical recommendations and software code are provided to support the application of SACE estimation in future research, particularly in CRTs.
Keywords:
Bayesian estimationalways-survivorscluster-randomized trialscounterfactual outcomesdeath truncationprincipal stratificationquality of lifesurvivor average causal effectMore Related Videos
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