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

doi:10.1214/21-AOAS1588

CSpace

	DATA-ADAPTIVE EFFICIENT ESTIMATION STRATEGIES FOR BIOMARKER STUDIES EMBEDDED IN RANDOMIZED TRIALS
	Zhang, Weiz 1; Zhang, Zhi wei 2; Troendle, James F.3; Liu, Aiyi 4
	2022-12-01
发表期刊	ANNALS OF APPLIED STATISTICS
ISSN	1932-6157
卷号	16 期号:4 页码:2250-2265
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.
关键词	Augmentation precision medicine semiparametric theory super learner two-phase sampling
DOI	10.1214/21-AOAS1588
收录类别	SCI
语种	英语
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
WOS类目	Statistics & Probability
WOS记录号	WOS:000868925900010
出版者	INST MATHEMATICAL STATISTICS-IMS
引用统计
文献类型	期刊论文
条目标识符	http://ir.amss.ac.cn/handle/2S8OKBNM/60765
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.Chinese Acad Sci, Acad Math & Syst Sci, Beijing, Peoples R China 2.NCI, NIH, Bethesda, MD USA 3.Natl Heart Lung & Blood Inst, NIH, Bethesda, MD USA 4.Eunice Kennedy Shriver Natl Inst Child Hlth & Huma, 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.