Targeted maximum likelihood estimation for dynamic treatment regimes in sequentially randomized controlled trials

International Journal of Biostatistics

Volumn 8, Issue 1, 2014, Pages

  Chaffee, Paul H ^a   Van Der Laan, Mark J ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

dynamic treatment regimes; estimation methods; semi parametric efficient estimation; sequential randomized controlled trials; targeted maximum likelihood estimation

Indexed keywords

ARTICLE; CONCEPTUAL FRAMEWORK; HUMAN; MAXIMUM LIKELIHOOD METHOD; PHARMACEUTICAL CARE; RANDOMIZED CONTROLLED TRIAL (TOPIC); STATISTICAL BIAS; STATISTICAL MODEL; TIME; BIOSTATISTICS; COMPUTER SIMULATION; EPIDEMIOLOGY; MATHEMATICAL PHENOMENA; METHODOLOGY; STATISTICS;

BIAS (EPIDEMIOLOGY); BIOSTATISTICS; CAUSALITY; COMPUTER SIMULATION; HUMANS; LIKELIHOOD FUNCTIONS; MATHEMATICAL CONCEPTS; MODELS, STATISTICAL; RANDOMIZED CONTROLLED TRIALS AS TOPIC; TIME FACTORS;

EID: 84907658657     PISSN: None     EISSN: 15574679     Source Type: Journal    
DOI: 10.1515/1557-4679.1406     Document Type: Article

References (29)

1. Bembom, O. and M. van der Laan (2007): "Statistical methods for analyzing sequentially randomized trials, " Journal of the National Cancer Institute, 99, 1577-1582.
2. Bembom, O. and M. van der Laan (2008): "Data-adaptive selection of the truncation level for inverse-probability-of-treatment-weighted estimators, " Berkeley Division of Biostatistics Working Paper Series. Working Paper 230.
3. Chaffee, P. and M. J. van der Laan (2011): "Targeted minimum loss based estimation based on directly solving the efficient influence curve equation, " U.C. Berkeley Division of Biostatistics Working Paper Series, Working Paper 287.
4. Chaffee, P. and M. J. van der Laan (2012): "Discussion of evaluation of viable dynamic treatment regimes in a sequentially randomized trial of advanced prostate cancer, by Wang et al. 2012, " Journal of the American Statistical Association.
5. Gruber, S. and M. J. van der Laan (2010): "A targeted maximum likelihood estimator of a causal effect on a bounded continuous outcome, " The International Journal of Biostatistics, 6.
6. Guo, X. and A. Tsiatis (2005): "A weighted risk set estimator for survival distributions in two-stage randomization designs with censored survival data, " International Journal of Biostatistics, 1.
7. Laber, E., M. Qian, D. Lizotte, and S. Murphy (2009): "Statistical inference in dynamic treatment regimes, " Revision of Univ. of Michigan, Statistics Department Technical Report 506.
8. Lunceford, J., M. Davidian, and A. Tsiatis (2002): "Estimation of survival distributions of treatment policies in two-stage randomization designs in clinical trials, " Biometrics, 58, 48-57.
9. Miyahara, S. and A. Wahed (2010): "Weighted Kaplan-Meier estimators for twostage treatment regimes, " Statistics in Medicine, 29, 2581-2591.
10. Moodie, E., R. Platt, and M. Kramer (2009): "Estimating response-maximized decision rules with applications to breastfeeding, " Journal of the American Statistical Association, 104, 155-165.
11. Murphy, S., M. van der Laan, and J. Robins (2001): "Marginal mean models for dynamic regimes, " Journal of the American Statistical Association, 6, 1410-1423.
12. Orellana, L., A. Rotnitzky, and J. Robins (2010): "Dynamic regime marginal structural mean models for estimation of optimal dynamic treatment regimes, " International Journal of Biostatistics, 6.
13. Pearl, J. (2000): Causality: Models, Reasoning and Inference, Cambridge: Cambridge University Press.
14. Petersen, M., Y. Wang, M. van der Laan, D. Guzman, E. Riley, and D. Bangsberg (2007): "Pillbox organizers are associated with improved adherence to HIV antiretroviral therapy and viral suppression: a marginal structural model analysis, " Clinical Infectious Diseases, 45, 908-15.
15. Porter, K. E., S. Gruber, M. J. van der Laan, and J. S. Sekhon (2011): "The relative performance of targeted maximum likelihood estimators, " International Journal of Biostatistics, 7.
16. Robins, J. (1986): "A new approach to causal inference in mortality studies with sustained exposure periods'application to control of the healthy worker survivor effect, " Mathematical Modeling, 7, 1393-1512.
17. Stitelman, O. M., V. D. Gruttola, and M. J. van der Laan (2011): "A general implementation of TMLE for longitudinal data applied to causal inference in survival analysis, " U.C. Berkeley Division of Biostatistics Working Paper Series, Working Paper 281.
18. Stitelman, O. M. and M. J. van der Laan (2010): "Collaborative targeted maximum likelihood for time to event data, " The International Journal of Biostatistics, 6.
19. Tunis, S., D. Faries, and Nyhuis, A.W. (2006): "Cost-effectiveness of olanzapine as first-line treatment for schizophrenia: results from a randomize, openlabel, 1-year trial, " Value Health, 9, 77-89.
20. van der Laan, M. and M. Petersen (2007): "Causal effect models for realistic individualized treatment and intention to treat rules, " The International Journal of Biostatistics, 3.
21. van der Laan, M. and J. Robins (2003): Unified methods for Censored Longitudinal Data and Causality, New York: Springer Verlag.
22. van der Laan, M. and S. Rose (2011): Targeted Learning: Causal Inference for Observational and Experimental Data, Berlin Heidelberg New York: Springer.
23. van der Laan, M. and D. Rubin (2006): "Targeted maximum likelihood learning, " The International Journal of Biostatistics, 2.
24. van der Laan, M. J. (2010a): "Targeted maximum likelihood based causal inference: Part I, " The International Journal of Biostatistics, 6.
25. van der Laan, M. J. (2010b): "Targeted maximum likelihood based causal inference: Part II, " The International Journal of Biostatistics, 6.
26. van der Laan, M. J., E. C. Polley, and A. E. Hubbard (2007): "Super learner, " U.C. Berkeley Division of Biostatistics Working Paper Series, Working paper 222, http://www.bepress.com/ucbbiostat/paper222.
27. Wahed, A. and A. Tsiatis (2004): "Optimal estimator for the survival distribution and related quantities for treatment policies in two-stage randomization designs in clinical trials, " Biometrics, 60, 124-133.
28. Wahed, A. and A. Tsiatis (2006): "Semi-parametric efficient estimation of the survival distribution for treatment policies in two-stage randomization designs in clinical trials with censored data, " Biometrika, 60, 147-161.
29. Wang, L., A. Rotnitzky, X. Lin, R. E. Millikan, and P. F. Thall (2012): "Evaluation of viable dynamic treatment regimes in a sequentially randomized trial of advanced prostate cancer, " Journal of the American Statistical Association, forthcoming.