Literature DB >> 23658214

The balanced survivor average causal effect.

Tom Greene, Marshall Joffe, Bo Hu, Liang Li, Ken Boucher.   

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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Year:  2013        PMID: 23658214     DOI: 10.1515/ijb-2012-0013

Source DB:  PubMed          Journal:  Int J Biostat        ISSN: 1557-4679            Impact factor:   0.968


  3 in total

1.  Power and sample size calculations for the Wilcoxon-Mann-Whitney test in the presence of death-censored observations.

Authors:  Roland A Matsouaka; Rebecca A Betensky
Journal:  Stat Med       Date:  2014-11-13       Impact factor: 2.373

2.  simcausal R Package: Conducting Transparent and Reproducible Simulation Studies of Causal Effect Estimation with Complex Longitudinal Data.

Authors:  Oleg Sofrygin; Mark J van der Laan; Romain Neugebauer
Journal:  J Stat Softw       Date:  2017-10-16       Impact factor: 6.440

3.  A comparison of methods to estimate the survivor average causal effect in the presence of missing data: a simulation study.

Authors:  Myra B McGuinness; Jessica Kasza; Amalia Karahalios; Robyn H Guymer; Robert P Finger; Julie A Simpson
Journal:  BMC Med Res Methodol       Date:  2019-12-03       Impact factor: 4.615

  3 in total

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