A threshold sample-enrichment approach in a clinical trial with heterogeneous subpopulations

doi:10.1177/1740774510378695

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	A threshold sample-enrichment approach in a clinical trial with heterogeneous subpopulations
	Liu, Aiyi 1; Liu, Chunling 1; Li, Qizhai2 ; Yu, Kai F.1; Yuan, Vivian W.3
	2010-10-01
发表期刊	CLINICAL TRIALS
ISSN	1740-7745
卷号	7 期号:5 页码:537-545
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
DOI	10.1177/1740774510378695
语种	英语
WOS研究方向	Research & Experimental Medicine
WOS类目	Medicine, Research & Experimental
WOS记录号	WOS:000282089800004
出版者	SAGE PUBLICATIONS LTD
引用统计
文献类型	期刊论文
条目标识符	http://ir.amss.ac.cn/handle/2S8OKBNM/9387
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.