Abstract: Statistical analysis of longitudinal outcomes is often complicated by the absence of observable values in patients who die prior to their scheduled measurement. In such cases, the longitudinal data are said to be “truncated by death” to emphasize that the longitudinal measurements are not simply missing, but are undefined after death. Recently, the truncation by death problem has been investigated using the framework of principal stratification to define the target estimand as the survivor average causal effect (SACE), which in the context of a two-group randomized clinical trial is the mean difference in the longitudinal outcome between the treatment and control groups for the principal stratum of always-survivors. The SACE is not identified without untestable assumptions. These assumptions have often been formulated in terms of a monotonicity constraint requiring that the treatment does not reduce survival in any patient, in conjunction with assumed values for mean differences in the longitudinal outcome between certain principal strata. In this paper, we introduce an alternative estimand, the balanced-SACE, which is defined as the average causal effect on the longitudinal outcome in a particular subset of the always-survivors that is balanced with respect to the potential survival times under the treatment and control. We propose a simple estimator of the balanced-SACE that compares the longitudinal outcomes between equivalent fractions of the longest surviving patients between the treatment and control groups and does not require a monotonicity assumption. We provide expressions for the large sample bias of the estimator, along with sensitivity analyses and strategies to minimize this bias. We consider statistical inference under a bootstrap resampling procedure.
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©2013 by Walter de Gruyter Berlin / Boston
Articles in the same Issue
- Masthead
- Masthead
- Research Articles
- Sensitivity Analysis for Causal Inference under Unmeasured Confounding and Measurement Error Problems
- Assessing the Causal Effect of Policies: An Example Using Stochastic Interventions
- Novel Point Estimation from a Semiparametric Ratio Estimator (SPRE): Long-Term Health Outcomes from Short-Term Linear Data, with Application to Weight Loss in Obesity
- Exact Nonparametric Confidence Bands for the Survivor Function
- Semiparametric Regression Analysis of Clustered Interval-Censored Failure Time Data with Informative Cluster Size
- A Weighting Analogue to Pair Matching in Propensity Score Analysis
- Alternative Monotonicity Assumptions for Improving Bounds on Natural Direct Effects
- Estimation of Risk Ratios in Cohort Studies with a Common Outcome: A Simple and Efficient Two-stage Approach
- Distance-Based Mapping of Disease Risk
- The Balanced Survivor Average Causal Effect
- Commentary
- Principal Stratification: A Broader Vision
Articles in the same Issue
- Masthead
- Masthead
- Research Articles
- Sensitivity Analysis for Causal Inference under Unmeasured Confounding and Measurement Error Problems
- Assessing the Causal Effect of Policies: An Example Using Stochastic Interventions
- Novel Point Estimation from a Semiparametric Ratio Estimator (SPRE): Long-Term Health Outcomes from Short-Term Linear Data, with Application to Weight Loss in Obesity
- Exact Nonparametric Confidence Bands for the Survivor Function
- Semiparametric Regression Analysis of Clustered Interval-Censored Failure Time Data with Informative Cluster Size
- A Weighting Analogue to Pair Matching in Propensity Score Analysis
- Alternative Monotonicity Assumptions for Improving Bounds on Natural Direct Effects
- Estimation of Risk Ratios in Cohort Studies with a Common Outcome: A Simple and Efficient Two-stage Approach
- Distance-Based Mapping of Disease Risk
- The Balanced Survivor Average Causal Effect
- Commentary
- Principal Stratification: A Broader Vision