IDEAS home Printed from https://ideas.repec.org/a/bpj/ijbist/v7y2011i1n19.html
   My bibliography  Save this article

Targeted Maximum Likelihood Estimation of Effect Modification Parameters in Survival Analysis

Author

Listed:
  • Stitelman Ori M
  • Wester C. William
  • De Gruttola Victor
  • van der Laan Mark J.

Abstract

The Cox proportional hazards model or its discrete time analogue, the logistic failure time model, posit highly restrictive parametric models and attempt to estimate parameters which are specific to the model proposed. These methods are typically implemented when assessing effect modification in survival analyses despite their flaws. The targeted maximum likelihood estimation (TMLE) methodology is more robust than the methods typically implemented and allows practitioners to estimate parameters that directly answer the question of interest. TMLE will be used in this paper to estimate two newly proposed parameters of interest that quantify effect modification in the time to event setting. These methods are then applied to the Tshepo study to assess if either gender or baseline CD4 level modify the effect of two cART therapies of interest, efavirenz (EFV) and nevirapine (NVP), on the progression of HIV. The results show that women tend to have more favorable outcomes using EFV while males tend to have more favorable outcomes with NVP. Furthermore, EFV tends to be favorable compared to NVP for individuals at high CD4 levels.

Suggested Citation

  • Stitelman Ori M & Wester C. William & De Gruttola Victor & van der Laan Mark J., 2011. "Targeted Maximum Likelihood Estimation of Effect Modification Parameters in Survival Analysis," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-34, March.
    • Handle: RePEc:bpj:ijbist:v:7:y:2011:i:1:n:19
    DOI: 10.2202/1557-4679.1307
    as

    Download full text from publisher

    File URL: https://doi.org/10.2202/1557-4679.1307
    Download Restriction: For access to full text, subscription to the journal or payment for the individual article is required.
    File URL: https://libkey.io/10.2202/1557-4679.1307?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. Stitelman Ori M & van der Laan Mark J., 2010. "Collaborative Targeted Maximum Likelihood for Time to Event Data," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-46, June.
    2. van der Laan Mark J. & Dudoit Sandrine & Keles Sunduz, 2004. "Asymptotic Optimality of Likelihood-Based Cross-Validation," Statistical Applications in Genetics and Molecular Biology, De Gruyter, vol. 3(1), pages 1-25, March.
    3. Porter Kristin E. & Gruber Susan & van der Laan Mark J. & Sekhon Jasjeet S., 2011. "The Relative Performance of Targeted Maximum Likelihood Estimators," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-34, August.
    4. van der Laan Mark J. & Rubin Daniel, 2006. "Targeted Maximum Likelihood Learning," The International Journal of Biostatistics, De Gruyter, vol. 2(1), pages 1-40, December.
    5. Gruber Susan & van der Laan Mark J., 2010. "A Targeted Maximum Likelihood Estimator of a Causal Effect on a Bounded Continuous Outcome," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-18, August.
    6. Heejung Bang & James M. Robins, 2005. "Doubly Robust Estimation in Missing Data and Causal Inference Models," Biometrics, The International Biometric Society, vol. 61(4), pages 962-973, December.
    7. van der Laan Mark J. & Gruber Susan, 2010. "Collaborative Double Robust Targeted Maximum Likelihood Estimation," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-71, May.
    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. Helene C. W. Rytgaard & Frank Eriksson & Mark J. van der Laan, 2023. "Estimation of time‐specific intervention effects on continuously distributed time‐to‐event outcomes by targeted maximum likelihood estimation," Biometrics, The International Biometric Society, vol. 79(4), pages 3038-3049, December.
    2. Stitelman Ori M. & De Gruttola Victor & van der Laan Mark J., 2012. "A General Implementation of TMLE for Longitudinal Data Applied to Causal Inference in Survival Analysis," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-39, September.
    3. van der Laan Mark J. & Gruber Susan, 2012. "Targeted Minimum Loss Based Estimation of Causal Effects of Multiple Time Point Interventions," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-41, May.
    4. Brooks Jordan & van der Laan Mark J. & Go Alan S., 2012. "Targeted Maximum Likelihood Estimation for Prediction Calibration," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-35, October.

    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. Stitelman Ori M. & De Gruttola Victor & van der Laan Mark J., 2012. "A General Implementation of TMLE for Longitudinal Data Applied to Causal Inference in Survival Analysis," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-39, September.
    2. Rose Sherri & van der Laan Mark J., 2011. "A Targeted Maximum Likelihood Estimator for Two-Stage Designs," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-21, March.
    3. Susan Gruber & Mark J. van der Laan, 2013. "An Application of Targeted Maximum Likelihood Estimation to the Meta-Analysis of Safety Data," Biometrics, The International Biometric Society, vol. 69(1), pages 254-262, March.
    4. Brooks Jordan & van der Laan Mark J. & Go Alan S., 2012. "Targeted Maximum Likelihood Estimation for Prediction Calibration," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-35, October.
    5. Chaffee Paul H. & van der Laan Mark J., 2012. "Targeted Maximum Likelihood Estimation for Dynamic Treatment Regimes in Sequentially Randomized Controlled Trials," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-32, June.
    6. van der Laan Mark J. & Gruber Susan, 2012. "Targeted Minimum Loss Based Estimation of Causal Effects of Multiple Time Point Interventions," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-41, May.
    7. Stitelman Ori M & van der Laan Mark J., 2010. "Collaborative Targeted Maximum Likelihood for Time to Event Data," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-46, June.
    8. Gruber Susan & van der Laan Mark J., 2010. "An Application of Collaborative Targeted Maximum Likelihood Estimation in Causal Inference and Genomics," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-31, May.
    9. Frölich, Markus & Huber, Martin & Wiesenfarth, Manuel, 2017. "The finite sample performance of semi- and non-parametric estimators for treatment effects and policy evaluation," Computational Statistics & Data Analysis, Elsevier, vol. 115(C), pages 91-102.
    10. Zheng Wenjing & van der Laan Mark J., 2012. "Targeted Maximum Likelihood Estimation of Natural Direct Effects," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-40, January.
    11. Mireille E. Schnitzer & Erica E.M. Moodie & Mark J. van der Laan & Robert W. Platt & Marina B. Klein, 2014. "Modeling the impact of hepatitis C viral clearance on end-stage liver disease in an HIV co-infected cohort with targeted maximum likelihood estimation," Biometrics, The International Biometric Society, vol. 70(1), pages 144-152, March.
    12. van der Laan Mark J. & Petersen Maya & Zheng Wenjing, 2013. "Estimating the Effect of a Community-Based Intervention with Two Communities," Journal of Causal Inference, De Gruyter, vol. 1(1), pages 83-106, June.
    13. Gruber, Susan & Laan, Mark van der, 2012. "tmle: An R Package for Targeted Maximum Likelihood Estimation," Journal of Statistical Software, Foundation for Open Access Statistics, vol. 51(i13).
    14. Radice Rosalba & Ramsahai Roland & Grieve Richard & Kreif Noemi & Sadique Zia & Sekhon Jasjeet S., 2012. "Evaluating treatment effectiveness in patient subgroups: a comparison of propensity score methods with an automated matching approach," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-45, August.
    15. Kara E. Rudolph & Mark J. Laan, 2017. "Robust estimation of encouragement design intervention effects transported across sites," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 79(5), pages 1509-1525, November.
    16. van der Laan Mark J., 2014. "Targeted Estimation of Nuisance Parameters to Obtain Valid Statistical Inference," The International Journal of Biostatistics, De Gruyter, vol. 10(1), pages 29-57, May.
    17. Waverly Wei & Maya Petersen & Mark J van der Laan & Zeyu Zheng & Chong Wu & Jingshen Wang, 2023. "Efficient targeted learning of heterogeneous treatment effects for multiple subgroups," Biometrics, The International Biometric Society, vol. 79(3), pages 1934-1946, September.
    18. Paul Frédéric Blanche & Anders Holt & Thomas Scheike, 2023. "On logistic regression with right censored data, with or without competing risks, and its use for estimating treatment effects," Lifetime Data Analysis: An International Journal Devoted to Statistical Methods and Applications for Time-to-Event Data, Springer, vol. 29(2), pages 441-482, April.
    19. Yiyi Huo & Yingying Fan & Fang Han, 2023. "On the adaptation of causal forests to manifold data," Papers 2311.16486, arXiv.org, revised Dec 2023.
    20. Gruber Susan & van der Laan Mark J., 2010. "A Targeted Maximum Likelihood Estimator of a Causal Effect on a Bounded Continuous Outcome," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-18, August.

    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:7:y:2011:i:1:n:19. 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.