DATA-ADAPTIVE EFFICIENT ESTIMATION STRATEGIES FOR BIOMARKER STUDIES EMBEDDED IN RANDOMIZED TRIALS

doi:10.1214/21-AOAS1588

	DATA-ADAPTIVE EFFICIENT ESTIMATION STRATEGIES FOR BIOMARKER STUDIES EMBEDDED IN RANDOMIZED TRIALS
	Zhang, Weiz 3; Zhang, Zhi wei 1; Troendle, James F.4; Liu, Aiyi 2
刊名	ANNALS OF APPLIED STATISTICS
	2022-12-01
卷号	16 期号:4 页码:2250-2265
关键词	Augmentation precision medicine semiparametric theory super learner two-phase sampling
ISSN号	1932-6157
DOI	10.1214/21-AOAS1588
英文摘要	Predictive and prognostic biomarkers are increasingly important in clini-cal research and practice. Biomarker studies are frequently embedded in ran-domized clinical trials with biospecimens collected at baseline and assayed for biomarkers, either in real time or retrospectively. This article proposes efficient estimation strategies for two study settings in terms of biomarker ascertainment: a complete-data setting in which the biomarker is measured for all subjects in the trial, and a two-phase sampling design in which the biomarker is measured retrospectively for a random subsample of subjects se-lected in an outcome-dependent fashion. In both settings, efficient estimating functions are characterized using semiparametric theory and approximated using data-adaptive machine learning methods, leading to estimators that are consistent, asymptotically normal and (approximately) efficient under gen-eral conditions. The proposed methods are evaluated in simulation studies and applied to real data from two biomarker studies, one in each setting.
资助项目	Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health ; National Heart, Lung and Blood Institute, National Institutes of Health ; National Natural Science Foundation of China[12001522] ; National Natural Science Foundation of China[72091212]
WOS研究方向	Mathematics
语种	英语
出版者	INST MATHEMATICAL STATISTICS-IMS
WOS记录号	WOS:000868925900010
内容类型	期刊论文
源URL	[http://ir.amss.ac.cn/handle/2S8OKBNM/60764]
专题	中国科学院数学与系统科学研究院
通讯作者	Zhang, Weiz
作者单位	1.NCI, NIH, Bethesda, MD USA 2.Eunice Kennedy Shriver Natl Inst Child Hlth & Huma, NIH, Bethesda, MD USA 3.Chinese Acad Sci, Acad Math & Syst Sci, Beijing, Peoples R China 4.Natl Heart Lung & Blood Inst, NIH, Bethesda, MD USA
推荐引用方式 GB/T 7714	Zhang, Weiz,Zhang, Zhi wei,Troendle, James F.,et al. DATA-ADAPTIVE EFFICIENT ESTIMATION STRATEGIES FOR BIOMARKER STUDIES EMBEDDED IN RANDOMIZED TRIALS[J]. ANNALS OF APPLIED STATISTICS,2022,16(4):2250-2265.
APA	Zhang, Weiz,Zhang, Zhi wei,Troendle, James F.,&Liu, Aiyi.(2022).DATA-ADAPTIVE EFFICIENT ESTIMATION STRATEGIES FOR BIOMARKER STUDIES EMBEDDED IN RANDOMIZED TRIALS.ANNALS OF APPLIED STATISTICS,16(4),2250-2265.
MLA	Zhang, Weiz,et al."DATA-ADAPTIVE EFFICIENT ESTIMATION STRATEGIES FOR BIOMARKER STUDIES EMBEDDED IN RANDOMIZED TRIALS".ANNALS OF APPLIED STATISTICS 16.4(2022):2250-2265.