A nonparametric method for value function guided subgroup identification via gradient tree boosting for censored survival data.

censored survival data gradient tree boosting nonparametric personalized medicine restricted mean survival time subgroup identification

Journal

Statistics in medicine
ISSN: 1097-0258
Titre abrégé: Stat Med
Pays: England
ID NLM: 8215016

Informations de publication

Date de publication:
10 12 2020
Historique:
received: 09 02 2020
revised: 08 06 2020
accepted: 09 07 2020
pubmed: 14 8 2020
medline: 22 6 2021
entrez: 14 8 2020
Statut: ppublish

Résumé

In randomized clinical trials with survival outcome, there has been an increasing interest in subgroup identification based on baseline genomic, proteomic markers, or clinical characteristics. Some of the existing methods identify subgroups that benefit substantially from the experimental treatment by directly modeling outcomes or treatment effect. When the goal is to find an optimal treatment for a given patient rather than finding the right patient for a given treatment, methods under the individualized treatment regime framework estimate an individualized treatment rule that would lead to the best expected clinical outcome as measured by a value function. Connecting the concept of value function to subgroup identification, we propose a nonparametric method that searches for subgroup membership scores by maximizing a value function that directly reflects the subgroup-treatment interaction effect based on restricted mean survival time. A gradient tree boosting algorithm is proposed to search for the individual subgroup membership scores. We conduct simulation studies to evaluate the performance of the proposed method and an application to an AIDS clinical trial is performed for illustration.

Identifiants

pubmed: 32786155
doi: 10.1002/sim.8714
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

4133-4146

Informations de copyright

© 2020 John Wiley & Sons, Ltd.

Références

Buzdar AU. Role of biologic therapy and chemotherapy in hormone receptor- and HER2- positive breast cancer. Ann Oncol. 2009;20(6):993-999.
Lipkovich I, Dmitrienko A, D'Agostino RB. Tutorial in biostatistics: data-driven subgroup identification and analysis in clinical trials. Stat Med. 2017;36(1):136-196.
Rubin DB. Estimating causal effects of treatments in randomized and nonrandomized studies. J Educ Psychol. 1974;66(5):688-701.
Imai K, Ratkovic M. Estimating treatment effect heterogeneity in randomized program evaluation. Ann Appl Stat. 2013;7(1):443-470.
Cai T, Tian L, Wong P, Wei LJ. Analysis of randomized comparative clinical trial data for personalized treatment selections. Biostatistics. 2011;12(2):270-282.
Zhao L, Tian L, Cai T, Claggett B, Wei L. Effectively selecting a target population for a future comparative study. J Am Stat Assoc. 2013;108(502):527-539.
Song X, Pepe MS. Evaluating markers for selecting a patient's treatment. Biometrics. 2004;60(4):874-883.
Huang Y, Gilbert PB, Janes H. Assessing treatment-selection markers using a potential outcomes framework. Biometrics. 2012;68(3):687-696.
Foster JC, Taylor JMC, Ruberg SJ. Subgroup identification from randomized clinical trial data. Stat Med. 2011;30(24):2867-2880.
Dusseldorp E, Conversano C, Van Os BJ. Combining an additive and tree-based regression model simultaneously: STIMA. J Comput Graph Stat. 2010;19(3):514-530.
Dixon DO, Simon R. Bayesian subset analysis. Biometrics. 1991;47(3):871-882.
Gu X, Yin G, Lee JJ. Bayesian two-step lasso strategy for biomarker selection in personalized medicine development for time-to-event endpoints. Contemp Clin Trials. 2013;36(2):642-650.
Lu M, Sadiq S, Feaster DJ, Ishwaran H. Estimating individual treatment effect in observational data using random forest methods. J Comput Graph Stat. 2018;27(1):209-219.
Sugasawa S, Noma H. Estimating individual treatment effects by gradient boosting trees. Stat Med. 2019;38(26):5146-5159.
Su X, Zhou T, Yan X, Fan J, Yang S. Interaction trees with censored survival data. Int J Biostat. 2008;4(1):2.
Su X, Tsai CL, Wang H, Nickerson DM, Li B. Subgroup analysis via recursive partitioning. J Mach Learn Res. 2009;10(5):141-158.
Loh WY, He X, Man M. A regression tree approach to identifying subgroups with differential treatment effects. Stat Med. 2015;34(11):1818-1833.
Zeileis A, Hothorn T, Hornik K. Model-based recursive partitioning. J Comput Graph Stat. 2008;17(2):492-514.
Dusseldorp E, Van Mechelen I. Qualitative interaction trees: a tool to identify qualitative treatment-subgroup interactions. Stat Med. 2014;33(2):219-237.
Tian L, Alizaden AA, Gentles AJ, Tibshirani R. A simple method for detecting interactions between a treatment and a large number of covariates. J Am Stat Assoc. 2014;109(508):1517-1532.
Chen G, Zhong H, Belousov A, Viswanath D. A PRIM approach to predictive-signature development for patient stratification. Stat Med. 2015;34(2):317-342.
Lipkovich I, Dmitrienko A, Denne J, Enas G. Subgroup identification based on differential effect search (SIDES): a recursive partitioning method for establishing response to treatment in patient subpopulations. Stat Med. 2011;30(21):2601-2621.
Qian M, Murphy SA. Performance guarantees for individualized treatment rules. Ann Stat. 2011;39(2):1180-1210.
Zhao Y, Zheng D, Rush AJ, Kosorok MR. Estimating individualized treatment rules using outcome weighted learning. J Am Stat Assoc. 2012;107(449):1106-1118.
Zhang B, Tsiatis AA, Davidian M, Zhang M, Laber EB. Estimating optimal treatment regimes from a classification perspective. Statistics. 2012;1(1):103-114.
Zhang B, Tsiatis AA, Laber EB, Davidian M. A robust method for estimating optimal treatment regimes. Biometrics. 2012;68(4):1010-1018.
Bai X, Tsiatis AA, Lu W, Song R. Optimal treatment regimes for survival endpoints using a locally-efficient doubly-robust estimator from a classification perspective. Lifetime Data Anal. 2017;23(4):585-604.
Cui Y, Zhu R, Kosorok M. Tree based weighted learning for estimating individualized treatment rules with censored data. Electron J Stat. 2017;11(2):3927-3953.
Mi X, Zou F, Zhu R. Bagging and deep learning in optimal individualized treatment rules. Biometrics. 2019;75(2):674-684.
Song R, Kosorok M, Zeng D, Zhao Y, Laber E, Yuan M. On sparse representation for optimal individualized treatment selection with penalized outcome weighted learning. Statistics. 2015;4(1):59-68.
Xu Y, Yu M, Zhao YQ, Li Q, Wang S, Shao J. Regularized outcome weighted subgroup identification for differential treatment effects. Biometrics. 2015;71(3):645-653.
Zhao YQ, Zeng D, Laber EB, Song R, Yuan M, Kosorok MR. Doubly robust learning for estimating individualized treatment with censored data. Biometrika. 2015;102(1):151-168.
Zhou X, Mayer-Hamblett N, Khan U, Kosorok MR. Residual weighted learning for estimating individualized treatment rules. J Am Stat Assoc. 2017;112(517):169-187.
Zhu R, Zhao YQ, Chen G, Ma S, Zhao H. Greedy outcome weighted tree learning of optimal personalized treatment rules. Biometrics. 2017;73(2):391-400.
Jiang R, Lu W, Song R, Davidian M. On estimation of optimal treatment regimes for maximizing t-year survival probability. J R Stat Soc Series B Stat Methodol. 2017;79(4):1165-1185.
Geng Y, Zhang HH, Lu W. On optimal treatment regimes selection for mean survival time. Stat Med. 2015;34(7):1169-1184.
Qi Z, Liu D, Fu H, Liu Y. Multi-armed angle-based direct learning for estimating optimal individualized treatment rules with various outcomes. J Am Stat Assoc. 2020;115(530):678-691.
Fu H, Zhou J, Faries DE. Estimating optimal treatment regimes via subgroup identification in randomized control trials and observational studies. Stat Med. 2016;35(19):3285-3302.
Karrison T. Restricted mean life with adjustment for covariates. J Am Stat Assoc. 1987;82(400):1169-1176.
Royston P, Parmar M. The use of restricted mean survival time to estimate the treatment effect in randomized clinical trials when the proportional hazards assumption is in doubt. J Am Stat Assoc. 2011;30(19):2409-2421.
Uno H, Claggett B, Tian L, et al. Moving beyond the hazard ratio in quantifying the between-group difference in survival analysis. J Clin Oncol. 2014;32(22):2380-2385.
Nelson W. Hazard plotting for incomplete failure data. J Qual Technol. 1969;1(1):27-52.
Friedman JH. Greedy function approximation: a gradient boosting machine. Ann Stat. 2001;29(5):1189-1232.
Chen T, Guestrin C. XGBoost: a scalable tree boosting system. Paper presented at: Proceedings of the 22nd ACM SIGKDD International Conference on Knowledge Discovery and Data Mining; 2016; San Francisco, CA.
Friedman J, Hastie T, Tibshirani R. Regularization paths for generalized linear models via coordinate descent. J Stat Softw. 2010;33(1):1-22.
Ishwaran H, Kogalur UB, Blackstone EH, Lauer MS. Random survival forests. Ann Appl Stat. 2008;2(3):841-860.
Breiman L. Random forests. Mach Learn. 2001;45(1):5-32.
Hammer SM, Katzenstein DA, Hughes MD, et al. A trial comparing nucleoside monotherapy with combination therapy in HIV-infected adults with CD4 cell counts from 200 to 500 per cubic millimeter. AIDS Clinical Trials Group study 175 study team. N Engl J Med. 1996;335(15):1081-1090.

Auteurs

Pingye Zhang (P)

Biostatistics and Research Decision Sciences, MRL, Merck & Co., Inc., Rahway, New Jersey, USA.

Junshui Ma (J)

Biostatistics and Research Decision Sciences, MRL, Merck & Co., Inc., Rahway, New Jersey, USA.

Xinqun Chen (X)

Biostatistics and Research Decision Sciences, MRL, Merck & Co., Inc., Rahway, New Jersey, USA.

Yue Shentu (Y)

Biostatistics and Research Decision Sciences, MRL, Merck & Co., Inc., Rahway, New Jersey, USA.

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