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Nonlinear predictive directions in clinical trials

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  • Cho, Youngjoo
  • Zhan, Xiang
  • Ghosh, Debashis

Abstract

In many clinical trials, individuals in different subgroups may experience differential treatment effects. This leads to the need to consider individualized differences in treatment benefit. The general concept of predictive directions, which are risk scores motivated by potential outcomes considerations, is introduced. These techniques borrow heavily from the literature from sufficient dimension reduction (SDR) and causal inference. Initially directions assuming an idealized complete data structure are formulated. Then a new connection between SDR and kernel machine methodology for detection of treatment-covariate interactions is developed. Simulation studies and a real data analysis from AIDS Clinical Trials Group (ACTG) 175 data show the utility of the proposed approach.

Suggested Citation

  • Cho, Youngjoo & Zhan, Xiang & Ghosh, Debashis, 2022. "Nonlinear predictive directions in clinical trials," Computational Statistics & Data Analysis, Elsevier, vol. 174(C).
  • Handle: RePEc:eee:csdana:v:174:y:2022:i:c:s0167947322000561
    DOI: 10.1016/j.csda.2022.107476
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    References listed on IDEAS

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    1. Nicolai Meinshausen & Peter Bühlmann, 2010. "Stability selection," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 72(4), pages 417-473, September.
    2. Yingcun Xia & Howell Tong & W. K. Li & Li‐Xing Zhu, 2002. "An adaptive estimation of dimension reduction space," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 64(3), pages 363-410, August.
    3. Kehl, Victoria & Ulm, Kurt, 2006. "Responder identification in clinical trials with censored data," Computational Statistics & Data Analysis, Elsevier, vol. 50(5), pages 1338-1355, March.
    4. Lan Wang & Yu Zhou & Rui Song & Ben Sherwood, 2018. "Quantile-Optimal Treatment Regimes," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 113(523), pages 1243-1254, July.
    5. Stefan Wager & Susan Athey, 2018. "Estimation and Inference of Heterogeneous Treatment Effects using Random Forests," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 113(523), pages 1228-1242, July.
    6. Wei Luo & Yeying Zhu & Debashis Ghosh, 2017. "On estimating regression-based causal effects using sufficient dimension reduction," Biometrika, Biometrika Trust, vol. 104(1), pages 51-65.
    7. Ghosh, Debashis, 2011. "Propensity score modelling in observational studies using dimension reduction methods," Statistics & Probability Letters, Elsevier, vol. 81(7), pages 813-820, July.
    8. Youngjoo Cho & Debashis Ghosh, 2021. "Quantile-Based Subgroup Identification for Randomized Clinical Trials," Statistics in Biosciences, Springer;International Chinese Statistical Association, vol. 13(1), pages 90-128, April.
    9. Tianxi Cai & Giulia Tonini & Xihong Lin, 2011. "Kernel Machine Approach to Testing the Significance of Multiple Genetic Markers for Risk Prediction," Biometrics, The International Biometric Society, vol. 67(3), pages 975-986, September.
    10. Dawei Liu & Xihong Lin & Debashis Ghosh, 2007. "Semiparametric Regression of Multidimensional Genetic Pathway Data: Least-Squares Kernel Machines and Linear Mixed Models," Biometrics, The International Biometric Society, vol. 63(4), pages 1079-1088, December.
    11. Yuanyuan Shen & Tianxi Cai, 2016. "Identifying predictive markers for personalized treatment selection," Biometrics, The International Biometric Society, vol. 72(4), pages 1017-1025, December.
    12. Qiang Wu & Feng Liang & Sayan Mukherjee, 2013. "Kernel Sliced Inverse Regression: Regularization and Consistency," Abstract and Applied Analysis, Hindawi, vol. 2013, pages 1-11, July.
    13. Su Xiaogang & Zhou Tianni & Yan Xin & Fan Juanjuan & Yang Song, 2008. "Interaction Trees with Censored Survival Data," The International Journal of Biostatistics, De Gruyter, vol. 4(1), pages 1-28, January.
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