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Targeted Maximum Likelihood Estimation of Effect Modification Parameters in Survival Analysis

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  • 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
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    References listed on IDEAS

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    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. 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.
    4. 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.
    5. 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.
    6. 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.
    7. 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.
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    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.

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