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Causal inference with outcomes truncated by death in multiarm studies

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  • Shanshan Luo
  • Wei Li
  • Yangbo He

Abstract

It is challenging to evaluate causal effects when the outcomes of interest suffer from truncation‐by‐death in many clinical studies; that is, outcomes cannot be observed if patients die before the time of measurement. To address this problem, it is common to consider average treatment effects by principal stratification, for which, the identifiability results and estimation methods with a binary treatment have been established in previous literature. However, in multiarm studies with more than two treatment options, estimation of causal effects becomes more complicated and requires additional techniques. In this article, we consider identification, estimation, and bounds of causal effects with multivalued ordinal treatments and the outcomes subject to truncation‐by‐death. We define causal parameters of interest in this setting and show that they are identifiable either using some auxiliary variable or based on linear model assumption. We then propose a semiparametric method for estimating the causal parameters and derive their asymptotic results. When the identification conditions are invalid, we derive sharp bounds of the causal effects by use of covariates adjustment. Simulation studies show good performance of the proposed estimator. We use the estimator to analyze the effects of a four‐level chronic toxin on fetal developmental outcomes such as birth weight in rats and mice, with data from a developmental toxicity trial conducted by the National Toxicology Program. Data analyses demonstrate that a high dose of the toxin significantly reduces the weights of pups.

Suggested Citation

  • Shanshan Luo & Wei Li & Yangbo He, 2023. "Causal inference with outcomes truncated by death in multiarm studies," Biometrics, The International Biometric Society, vol. 79(1), pages 502-513, March.
  • Handle: RePEc:bla:biomet:v:79:y:2023:i:1:p:502-513
    DOI: 10.1111/biom.13554
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    References listed on IDEAS

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    1. Hansen, Lars Peter, 1982. "Large Sample Properties of Generalized Method of Moments Estimators," Econometrica, Econometric Society, vol. 50(4), pages 1029-1054, July.
    2. Constantine E. Frangakis & Donald B. Rubin, 2002. "Principal Stratification in Causal Inference," Biometrics, The International Biometric Society, vol. 58(1), pages 21-29, March.
    3. Peng Ding & Jiannan Lu, 2017. "Principal stratification analysis using principal scores," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 79(3), pages 757-777, June.
    4. Peter B. Gilbert & Ronald J. Bosch & Michael G. Hudgens, 2003. "Sensitivity Analysis for the Assessment of Causal Vaccine Effects on Viral Load in HIV Vaccine Trials," Biometrics, The International Biometric Society, vol. 59(3), pages 531-541, September.
    5. Leonardo Grilli & Fabrizia Mealli, 2008. "Nonparametric Bounds on the Causal Effect of University Studies on Job Opportunities Using Principal Stratification," Journal of Educational and Behavioral Statistics, , vol. 33(1), pages 111-130, March.
    6. Lee, Keunbaik & Daniels, Michael J. & Sargent, Daniel J., 2010. "Causal Effects of Treatments for Informative Missing Data due to Progression/Death," Journal of the American Statistical Association, American Statistical Association, vol. 105(491), pages 912-929.
    7. Imai, Kosuke, 2008. "Sharp bounds on the causal effects in randomized experiments with "truncation-by-death"," Statistics & Probability Letters, Elsevier, vol. 78(2), pages 144-149, February.
    8. Douglas Hayden & Donna K. Pauler & David Schoenfeld, 2005. "An Estimator for Treatment Comparisons among Survivors in Randomized Trials," Biometrics, The International Biometric Society, vol. 61(1), pages 305-310, March.
    9. Bryan E. Shepherd & Peter B. Gilbert & Yannis Jemiai & Andrea Rotnitzky, 2006. "Sensitivity Analyses Comparing Outcomes Only Existing in a Subset Selected Post-Randomization, Conditional on Covariates, with Application to HIV Vaccine Trials," Biometrics, The International Biometric Society, vol. 62(2), pages 332-342, June.
    10. Linbo Wang & Xiao-Hua Zhou & Thomas S. Richardson, 2017. "Identification and estimation of causal effects with outcomes truncated by death," Biometrika, Biometrika Trust, vol. 104(3), pages 597-612.
    11. Frangakis, Constantine E. & Brookmeyer, Ronald S. & Varadhan, Ravi & Safaeian, Mahboobeh & Vlahov, David & Strathdee, Steffanie A., 2004. "Methodology for Evaluating a Partially Controlled Longitudinal Treatment Using Principal Stratification, With Application to a Needle Exchange Program," Journal of the American Statistical Association, American Statistical Association, vol. 99, pages 239-249, January.
    12. Pearl Judea, 2011. "Principal Stratification -- a Goal or a Tool?," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-13, March.
    13. Linbo Wang & Thomas S. Richardson & Xiao-Hua Zhou, 2017. "Causal analysis of ordinal treatments and binary outcomes under truncation by death," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 79(3), pages 719-735, June.
    14. Dustin M. Long & Michael G. Hudgens, 2013. "Sharpening Bounds on Principal Effects with Covariates," Biometrics, The International Biometric Society, vol. 69(4), pages 812-819, December.
    15. David S. Lee, 2009. "Training, Wages, and Sample Selection: Estimating Sharp Bounds on Treatment Effects," The Review of Economic Studies, Review of Economic Studies Ltd, vol. 76(3), pages 1071-1102.
    16. Imbens,Guido W. & Rubin,Donald B., 2015. "Causal Inference for Statistics, Social, and Biomedical Sciences," Cambridge Books, Cambridge University Press, number 9780521885881, October.
    17. Michael R. Elliott & Marshall M. Joffe & Zhen Chen, 2006. "A Potential Outcomes Approach to Developmental Toxicity Analyses," Biometrics, The International Biometric Society, vol. 62(2), pages 352-360, June.
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