DATA-ADAPTIVE EFFICIENT ESTIMATION STRATEGIES FOR BIOMARKER STUDIES EMBEDDED IN RANDOMIZED TRIALS
文献类型：期刊论文
| 作者 | Zhang, Weiz3; Zhang, Zhi wei1; Troendle, James F.4; Liu, Aiyi2 |
| 刊名 | ANNALS OF APPLIED STATISTICS
 |
| 出版日期 | 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 |
| WOS记录号 | WOS:000868925900010 |
| 出版者 | INST MATHEMATICAL STATISTICS-IMS |
| 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. |
入库方式： OAI收割
来源：数学与系统科学研究院
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