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Estimating individualized treatment rules for ordinal treatments

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  • Jingxiang Chen
  • Haoda Fu
  • Xuanyao He
  • Michael R. Kosorok
  • Yufeng Liu

Abstract

Precision medicine is an emerging scientific topic for disease treatment and prevention that takes into account individual patient characteristics. It is an important direction for clinical research, and many statistical methods have been proposed recently. One of the primary goals of precision medicine is to obtain an optimal individual treatment rule (ITR), which can help make decisions on treatment selection according to each patient's specific characteristics. Recently, outcome weighted learning (OWL) has been proposed to estimate such an optimal ITR in a binary treatment setting by maximizing the expected clinical outcome. However, for ordinal treatment settings, such as individualized dose finding, it is unclear how to use OWL. In this article, we propose a new technique for estimating ITR with ordinal treatments. In particular, we propose a data duplication technique with a piecewise convex loss function. We establish Fisher consistency for the resulting estimated ITR under certain conditions, and obtain the convergence and risk bound properties. Simulated examples and an application to a dataset from a type 2 diabetes mellitus observational study demonstrate the highly competitive performance of the proposed method compared to existing alternatives.

Suggested Citation

  • Jingxiang Chen & Haoda Fu & Xuanyao He & Michael R. Kosorok & Yufeng Liu, 2018. "Estimating individualized treatment rules for ordinal treatments," Biometrics, The International Biometric Society, vol. 74(3), pages 924-933, September.
    • Handle: RePEc:bla:biomet:v:74:y:2018:i:3:p:924-933
    DOI: 10.1111/biom.12865
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    References listed on IDEAS

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    1. Guanhua Chen & Donglin Zeng & Michael R. Kosorok, 2016. "Personalized Dose Finding Using Outcome Weighted Learning," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 111(516), pages 1509-1521, October.
    2. Yingqi Zhao & Donglin Zeng & A. John Rush & Michael R. Kosorok, 2012. "Estimating Individualized Treatment Rules Using Outcome Weighted Learning," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 107(499), pages 1106-1118, September.
    3. Baqun Zhang & Anastasios A. Tsiatis & Eric B. Laber & Marie Davidian, 2012. "A Robust Method for Estimating Optimal Treatment Regimes," Biometrics, The International Biometric Society, vol. 68(4), pages 1010-1018, December.
    4. Yaoyao Xu & Menggang Yu & Ying‐Qi Zhao & Quefeng Li & Sijian Wang & Jun Shao, 2015. "Regularized outcome weighted subgroup identification for differential treatment effects," Biometrics, The International Biometric Society, vol. 71(3), pages 645-653, September.
    5. E. B. Laber & Y. Q. Zhao, 2015. "Tree-based methods for individualized treatment regimes," Biometrika, Biometrika Trust, vol. 102(3), pages 501-514.
    6. Xin Zhou & Nicole Mayer-Hamblett & Umer Khan & Michael R. Kosorok, 2017. "Residual Weighted Learning for Estimating Individualized Treatment Rules," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 112(517), pages 169-187, January.
    7. Lihui Zhao & Lu Tian & Tianxi Cai & Brian Claggett & L. J. Wei, 2013. "Effectively Selecting a Target Population for a Future Comparative Study," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 108(502), pages 527-539, June.
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    Cited by:

    1. Yizhe Xu & Tom H. Greene & Adam P. Bress & Brian C. Sauer & Brandon K. Bellows & Yue Zhang & William S. Weintraub & Andrew E. Moran & Jincheng Shen, 2022. "Estimating the optimal individualized treatment rule from a cost‐effectiveness perspective," Biometrics, The International Biometric Society, vol. 78(1), pages 337-351, March.
    2. Yizhe Xu & Tom H. Greene & Adam P. Bress & Brandon K. Bellows & Yue Zhang & Zugui Zhang & Paul Kolm & William S. Weintraub & Andrew S. Moran & Jincheng Shen, 2022. "An Efficient Approach for Optimizing the Cost-effective Individualized Treatment Rule Using Conditional Random Forest," Papers 2204.10971, arXiv.org.
    3. Xinyang Huang & Jin Xu, 2020. "Estimating individualized treatment rules with risk constraint," Biometrics, The International Biometric Society, vol. 76(4), pages 1310-1318, December.
    4. Weibin Mo & Yufeng Liu, 2022. "Efficient learning of optimal individualized treatment rules for heteroscedastic or misspecified treatment‐free effect models," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 84(2), pages 440-472, April.
    5. Crystal T. Nguyen & Daniel J. Luckett & Anna R. Kahkoska & Grace E. Shearrer & Donna Spruijt‐Metz & Jaimie N. Davis & Michael R. Kosorok, 2020. "Estimating individualized treatment regimes from crossover designs," Biometrics, The International Biometric Society, vol. 76(3), pages 778-788, September.
    6. Zhen Li & Jie Chen & Eric Laber & Fang Liu & Richard Baumgartner, 2023. "Optimal Treatment Regimes: A Review and Empirical Comparison," International Statistical Review, International Statistical Institute, vol. 91(3), pages 427-463, December.

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