IDEAS home Printed from https://ideas.repec.org/a/bpj/ijbist/v8y2012i1n12.html
   My bibliography  Save this article

The Large Sample Bounds on the Principal Strata Effect with Application to a Prostate Cancer Prevention Trial

Author

Listed:
  • Chiba Yasutaka

    (Kinki University School of Medicine)

Abstract

Issues of post-randomization selection bias and truncation-by-death can arise in randomized clinical trials; for example, in a cancer prevention trial, an outcome such as cancer severity is undefined for individuals who do not develop cancer. Restricting analysis to a subpopulation selected after randomization can give rise to biased outcome comparisons. One approach to deal with such issues is to consider the principal strata effect (PSE, or equally, the survivor average causal effect). PSE is defined as the effect of treatment on the outcome among the subpopulation that would have been selected under either treatment arm. Unfortunately, the PSE cannot generally be estimated without the identifying assumptions; however, the bounds can be derived using a deterministic causal model. In this paper, we propose a number of assumptions for deriving the bounds with narrow width. The assumptions and bounds, which differ from those introduced by Zhang and Rubin (2003), are illustrated using data from a randomized prostate cancer prevention trial.

Suggested Citation

  • Chiba Yasutaka, 2012. "The Large Sample Bounds on the Principal Strata Effect with Application to a Prostate Cancer Prevention Trial," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-19, May.
  • Handle: RePEc:bpj:ijbist:v:8:y:2012:i:1:n:12
    DOI: 10.1515/1557-4679.1365
    as

    Download full text from publisher

    File URL: https://doi.org/10.1515/1557-4679.1365
    Download Restriction: For access to full text, subscription to the journal or payment for the individual article is required.

    File URL: https://libkey.io/10.1515/1557-4679.1365?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    As the access to this document is restricted, you may want to search for a different version of it.

    References listed on IDEAS

    as
    1. Shepherd, Bryan E. & Redman, Mary W. & Ankerst, Donna P., 2008. "Does Finasteride Affect the Severity of Prostate Cancer? A Causal Sensitivity Analysis," Journal of the American Statistical Association, American Statistical Association, vol. 103(484), pages 1392-1404.
    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. 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.
    4. A. Mattei & F. Mealli, 2007. "Application of the Principal Stratification Approach to the Faenza Randomized Experiment on Breast Self-Examination," Biometrics, The International Biometric Society, vol. 63(2), pages 437-446, June.
    5. Constantine E. Frangakis & Donald B. Rubin & Ming-Wen An & Ellen MacKenzie, 2007. "Principal Stratification Designs to Estimate Input Data Missing Due to Death," Biometrics, The International Biometric Society, vol. 63(3), pages 641-649, September.
    6. Hudgens, Michael G. & Halloran, M. Elizabeth, 2006. "Causal Vaccine Effects on Binary Postinfection Outcomes," Journal of the American Statistical Association, American Statistical Association, vol. 101, pages 51-64, March.
    7. Arvid Sjölander & Keith Humphreys & Stijn Vansteelandt & Rino Bellocco & Juni Palmgren, 2009. "Sensitivity Analysis for Principal Stratum Direct Effects, with an Application to a Study of Physical Activity and Coronary Heart Disease," Biometrics, The International Biometric Society, vol. 65(2), pages 514-520, June.
    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.
    Full references (including those not matched with items on IDEAS)

    Citations

    Citations are extracted by the CitEc Project, subscribe to its RSS feed for this item.
    as


    Cited by:

    1. Fan Yang & Dylan S. Small, 2016. "Using post-outcome measurement information in censoring-by-death problems," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 78(1), pages 299-318, January.
    2. Gilbert Peter B. & Blette Bryan S. & Shepherd Bryan E. & Hudgens Michael G., 2020. "Post-randomization Biomarker Effect Modification Analysis in an HIV Vaccine Clinical Trial," Journal of Causal Inference, De Gruyter, vol. 8(1), pages 54-69, January.
    3. Gilbert Peter B. & Blette Bryan S. & Hudgens Michael G. & Shepherd Bryan E., 2020. "Post-randomization Biomarker Effect Modification Analysis in an HIV Vaccine Clinical Trial," Journal of Causal Inference, De Gruyter, vol. 8(1), pages 54-69, January.

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. VanderWeele Tyler J, 2011. "Principal Stratification -- Uses and Limitations," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-14, July.
    2. 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.
    3. Dean Follmann & Michael P. Fay & Michael Proschan, 2009. "Chop-Lump Tests for Vaccine Trials," Biometrics, The International Biometric Society, vol. 65(3), pages 885-893, September.
    4. Bryan E. Shepherd & Peter B. Gilbert & Charles T. Dupont, 2011. "Sensitivity Analyses Comparing Time-to-Event Outcomes Only Existing in a Subset Selected Postrandomization and Relaxing Monotonicity," Biometrics, The International Biometric Society, vol. 67(3), pages 1100-1110, September.
    5. 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.
    6. Fan Yang & Dylan S. Small, 2016. "Using post-outcome measurement information in censoring-by-death problems," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 78(1), pages 299-318, January.
    7. Sjolander Arvid & Vansteelandt Stijn & Humphreys Keith, 2010. "A Principal Stratification Approach to Assess the Differences in Prognosis between Cancers Caused by Hormone Replacement Therapy and by Other Factors," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-37, June.
    8. James Robins & Andrea Rotnitzky & Stijn Vansteelandt, 2007. "Discussions," Biometrics, The International Biometric Society, vol. 63(3), pages 650-653, September.
    9. 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.
    10. Halloran M. Elizabeth & Hudgens Michael G., 2012. "Causal Inference for Vaccine Effects on Infectiousness," The International Journal of Biostatistics, De Gruyter, vol. 8(2), pages 1-40, January.
    11. Andrea Mercatanti & Fan Li, 2017. "Do debit cards decrease cash demand?: causal inference and sensitivity analysis using principal stratification," Journal of the Royal Statistical Society Series C, Royal Statistical Society, vol. 66(4), pages 759-776, August.
    12. Yannis Jemiai & Andrea Rotnitzky & Bryan E. Shepherd & Peter B. Gilbert, 2007. "Semiparametric estimation of treatment effects given base‐line covariates on an outcome measured after a post‐randomization event occurs," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 69(5), pages 879-901, November.
    13. 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.
    14. Brian L. Egleston & Robert G. Uzzo & Yu-Ning Wong, 2017. "Latent Class Survival Models Linked by Principal Stratification to Investigate Heterogenous Survival Subgroups Among Individuals With Early-Stage Kidney Cancer," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 112(518), pages 534-546, April.
    15. VanderWeele, Tyler J., 2008. "Simple relations between principal stratification and direct and indirect effects," Statistics & Probability Letters, Elsevier, vol. 78(17), pages 2957-2962, December.
    16. Arvid Sjölander & Keith Humphreys & Stijn Vansteelandt & Rino Bellocco & Juni Palmgren, 2009. "Sensitivity Analysis for Principal Stratum Direct Effects, with an Application to a Study of Physical Activity and Coronary Heart Disease," Biometrics, The International Biometric Society, vol. 65(2), pages 514-520, June.
    17. Gilbert Peter B. & Hudgens Michael G. & Wolfson Julian, 2011. "Commentary on "Principal Stratification -- a Goal or a Tool?" by Judea Pearl," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-15, September.
    18. Alessandra Mattei & Fabrizia Mealli & Barbara Pacini, 2014. "Identification of causal effects in the presence of nonignorable missing outcome values," Biometrics, The International Biometric Society, vol. 70(2), pages 278-288, June.
    19. Rachel Axelrod & Daniel Nevo, 2023. "A sensitivity analysis approach for the causal hazard ratio in randomized and observational studies," Biometrics, The International Biometric Society, vol. 79(3), pages 2743-2756, September.
    20. Dean Follmann, 2006. "Augmented Designs to Assess Immune Response in Vaccine Trials," Biometrics, The International Biometric Society, vol. 62(4), pages 1161-1169, December.

    More about this item

    Statistics

    Access and download statistics

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:bpj:ijbist:v:8:y:2012:i:1:n:12. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Peter Golla (email available below). General contact details of provider: https://www.degruyter.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.