@article{bai_tsiatis_lu_song_2017, title={Optimal treatment regimes for survival endpoints using locally-efficient doubly-robust estimator from a classification perspective}, volume={23}, ISSN={["1572-9249"]}, DOI={10.1007/s10985-016-9376-x}, abstractNote={A treatment regime at a single decision point is a rule that assigns a treatment, among the available options, to a patient based on the patient’s baseline characteristics. The value of a treatment regime is the average outcome of a population of patients if they were all treated in accordance to the treatment regime, where large values are desirable. The optimal treatment regime is a regime which results in the greatest value. Typically, the optimal treatment regime is estimated by positing a regression relationship for the outcome of interest as a function of treatment and baseline characteristics. However, this can lead to suboptimal treatment regimes when the regression model is misspecified. We instead consider value search estimators for the optimal treatment regime where we directly estimate the value for any treatment regime and then maximize this estimator over a class of regimes. For many studies the primary outcome of interest is survival time which is often censored. We derive a locally efficient, doubly robust, augmented inverse probability weighted complete case estimator for the value function with censored survival data and study the large sample properties of this estimator. The optimization is realized from a weighted classification perspective that allows us to use available off the shelf software. In some studies one treatment may have greater toxicity or side effects, thus we also consider estimating a quality adjusted optimal treatment regime that allows a patient to trade some additional risk of death in order to avoid the more invasive treatment.}, number={4}, journal={Lifetime Data Analysis}, author={Bai, X. and Tsiatis, A. and Lu, W. and Song, R.}, year={2017}, pages={585–604} } @article{bai_tsiatis_2016, title={A log rank type test in observational survival studies with stratified sampling}, volume={22}, ISSN={["1572-9249"]}, DOI={10.1007/s10985-015-9331-2}, abstractNote={In randomized clinical trials, the log rank test is often used to test the null hypothesis of the equality of treatment-specific survival distributions. In observational studies, however, the ordinary log rank test is no longer guaranteed to be valid. In such studies we must be cautious about potential confounders; that is, the covariates that affect both the treatment assignment and the survival distribution. In this paper, two cases were considered: the first is when it is believed that all the potential confounders are captured in the primary database, and the second case where a substudy is conducted to capture additional confounding covariates. We generalize the augmented inverse probability weighted complete case estimators for treatment-specific survival distribution proposed in Bai et al. (Biometrics 69:830–839, 2013) and develop the log rank type test in both cases. The consistency and double robustness of the proposed test statistics are shown in simulation studies. These statistics are then applied to the data from the observational study that motivated this research.}, number={2}, journal={LIFETIME DATA ANALYSIS}, author={Bai, Xiaofei and Tsiatis, Anastasios A.}, year={2016}, month={Apr}, pages={280–298} } @article{bai_liu_li_faries_2015, title={Adaptive truncated weighting for improving marginal structural model estimation of treatment effects informally censored by subsequent therapy}, volume={14}, ISSN={["1539-1612"]}, DOI={10.1002/pst.1719}, abstractNote={Randomized clinical trials are designed to estimate the direct effect of a treatment by randomly assigning patients to receive either treatment or control. However, in some trials, patients who discontinued their initial randomized treatment are allowed to switch to another treatment. Therefore, the direct treatment effect of interest may be confounded by subsequent treatment. Moreover, the decision on whether to initiate a second‐line treatment is typically made based on time‐dependent factors that may be affected by prior treatment history. Due to these time‐dependent confounders, traditional time‐dependent Cox models may produce biased estimators of the direct treatment effect. Marginal structural models (MSMs) have been applied to estimate causal treatment effects even in the presence of time‐dependent confounders. However, the occurrence of extremely large weights can inflate the variance of the MSM estimators. In this article, we proposed a new method for estimating weights in MSMs by adaptively truncating the longitudinal inverse probabilities. This method provides balance in the bias variance trade‐off when large weights are inevitable, without the ad hoc removal of selected observations. We conducted simulation studies to explore the performance of different methods by comparing bias, standard deviation, confidence interval coverage rates, and mean square error under various scenarios. We also applied these methods to a randomized, open‐label, phase III study of patients with nonsquamous non‐small cell lung cancer. Copyright © 2015 John Wiley & Sons, Ltd.}, number={6}, journal={PHARMACEUTICAL STATISTICS}, author={Bai, Xiaofei and Liu, Jingyi and Li, Li and Faries, Douglas}, year={2015}, pages={448–454} } @article{bai_tsiatis_sean m. o'brien_2013, title={Doubly-Robust Estimators of Treatment-Specific Survival Distributions in Observational Studies with Stratified Sampling}, volume={69}, ISSN={["1541-0420"]}, DOI={10.1111/biom.12076}, abstractNote={Summary}, number={4}, journal={BIOMETRICS}, author={Bai, Xiaofei and Tsiatis, Anastasios A. and Sean M. O'Brien}, year={2013}, month={Dec}, pages={830–839} }