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存在缺失观测值的II期临床试验中多重缓解率的估计

Estimation of multiple response rates in phase II clinical trials with missing observations.

作者信息

Chang Myron

机构信息

Division of Biostatistics, Department of Epidemiology and Health Policy Research, College of Medicine, University of Florida, Gainesville, Florida 32608, USA.

出版信息

J Biopharm Stat. 2009 Sep;19(5):791-802. doi: 10.1080/10543400903105182.

Abstract

Responses are often correlated in clinical trials. A patient who is not evaluable for a response may still provide some information to the response through his or her status on other responses. Under the assumption of missing at random, we propose the utilization of a self-consistent estimator. We show that the proposed estimators are more efficient than the conventional estimators by asymptotic relative efficiency and simulation study. An example from a Phase II clinical trial on children with chronic myelogenous leukemia is provided.

摘要

在临床试验中,反应往往是相关的。一个对反应不可评估的患者仍可能通过其在其他反应上的状态为反应提供一些信息。在随机缺失的假设下,我们提出使用一种自洽估计量。我们通过渐近相对效率和模拟研究表明,所提出的估计量比传统估计量更有效。文中给出了一项针对慢性粒细胞白血病儿童的II期临床试验的例子。

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