A threshold sample-enrichment approach in a clinical trial with heterogeneous subpopulations
文献类型：期刊论文
| 作者 | Liu, Aiyi1; Liu, Chunling1; Li, Qizhai2 ; Yu, Kai F.1; Yuan, Vivian W.3
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| 刊名 | CLINICAL TRIALS
 |
| 出版日期 | 2010-10-01 |
| 卷号 | 7期号:5页码:537-545 |
| ISSN号 | 1740-7745 |
| DOI | 10.1177/1740774510378695 |
| 英文摘要 | Background Large comparative clinical trials usually target a wide-range of patients population in which subgroups exist according to certain patients' characteristics. Often, scientific knowledge or existing empirical data support the assumption that patients' improvement is larger among certain subgroups than others. Such information can be used to design a more cost-effective clinical trial. Purpose The goal of the article is to use such information to design a more cost-effective clinical trial. Methods A two-stage sample-enrichment design strategy is proposed that begins with enrollment from certain subgroup of patients and allows the trial to be terminated for futility in that subgroup. Results Simulation studies show that the two-stage sample-enrichment strategy is cost-effective if indeed the null hypothesis of no treatment improvement is true, as also so illustrated with data from a completed trial of calcium to prevent preeclampsia. Limitations Feasibility of the proposed enrichment design relies on the knowledge prior to the start of the trial that certain patients can benefit more than others from the treatment. Prolonged accrual and longer-waited outcomes may hinder utilization of the proposed design. Conclusions The two-stage sample-enrichment approach borrows strength from treatment heterogeneity among target patients in a large-scale comparative clinical trial, and is more cost-effective if the treatment arms are indeed of no difference. Clinical Trials 2010; 7: 537-545. http://ctj.sagepub.com |
| WOS研究方向 | Research & Experimental Medicine |
| 语种 | 英语 |
| WOS记录号 | WOS:000282089800004 |
| 出版者 | SAGE PUBLICATIONS LTD |
| 源URL | [http://ir.amss.ac.cn/handle/2S8OKBNM/9386]  |
| 专题 | 系统科学研究所 |
| 通讯作者 | Liu, Aiyi |
| 作者单位 | 1.Eunice Kennedy Shriver Natl Inst Child Hlth & Hum, Biostat & Bioinformat Branch, Rockville, MD 20852 USA 2.Chinese Acad Sci, Acad Math & Syst Sci, Beijing, Peoples R China 3.US FDA, Ctr Drug Evaluat & Res, Silver Spring, MD USA |
| 推荐引用方式 GB/T 7714 | Liu, Aiyi,Liu, Chunling,Li, Qizhai,et al. A threshold sample-enrichment approach in a clinical trial with heterogeneous subpopulations[J]. CLINICAL TRIALS,2010,7(5):537-545. |
| APA | Liu, Aiyi,Liu, Chunling,Li, Qizhai,Yu, Kai F.,&Yuan, Vivian W..(2010).A threshold sample-enrichment approach in a clinical trial with heterogeneous subpopulations.CLINICAL TRIALS,7(5),537-545. |
| MLA | Liu, Aiyi,et al."A threshold sample-enrichment approach in a clinical trial with heterogeneous subpopulations".CLINICAL TRIALS 7.5(2010):537-545. |
入库方式： OAI收割
来源：数学与系统科学研究院
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