Covariate adjustment for two-sample treatment comparisons in randomized clinical trials: a principled yet flexible approach.
Covariate adjustment for two-sample treatment comparisons in randomized clinical trials: a principled yet flexible approach.
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DOI：
10.1002/sim.3113
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发表时间：
2008-10-15
影响因子：
2
通讯作者：
Lu, Xiaomin
中科院分区：
文献类型：
--
作者：
Tsiatis, Anastasios A.;Davidian, Marie;Zhang, Min;Lu, Xiaomin
关键词：
关于是否以及如何使用协变量调整分析来比较随机临床试验中的治疗方法存在相当多的争论。在此类试验中，通常会收集大量基线协变量信息，调整的目标之一是利用与结果相关的协变量来提高治疗效果估计的精度。然而，当所使用的协变量是事后选择时，人们通常会担心潜在的偏差；以及基于结果、协变量和治疗之间的关系模型进行调整的潜力，以邀请“钓鱼探险”来获得最显着的效果估计。通过诉诸半参数理论，我们自然而然地得到了所有治疗效果估计量的特征，以及有原则的、实际可行的协变量调整方法，这些方法产生了所需的效率增益，并允许识别和利用协变量关系，同时规避了通常的担忧。提出了在实践中实施的方法和策略。模拟研究和对 HIV 临床试验数据的应用证明了该技术相对于现有方法的性能。
There is considerable debate regarding whether and how covariate adjusted analyses should be used in the comparison of treatments in randomized clinical trials. Substantial baseline covariate information is routinely collected in such trials, and one goal of adjustment is to exploit covariates associated with outcome to increase precision of estimation of the treatment effect. However, concerns are routinely raised over the potential for bias when the covariates used are selected post hoc; and the potential for adjustment based on a model of the relationship between outcome, covariates, and treatment to invite a “fishing expedition” for that leading to the most dramatic effect estimate. By appealing to the theory of semiparametrics, we are led naturally to a characterization of all treatment effect estimators and to principled, practically-feasible methods for covariate adjustment that yield the desired gains in efficiency and that allow covariate relationships to be identified and exploited while circumventing the usual concerns. The methods and strategies for their implementation in practice are presented. Simulation studies and an application to data from an HIV clinical trial demonstrate the performance of the techniques relative to existing methods.
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影响因子：
158.5
作者：
Hammer, SM;Katzenstein, DA;Salgo, M
通讯作者：
Salgo, M
影响因子：
2.7
作者：
CASSEL, CM;SARNDAL, CE;WRETMAN, JH
通讯作者：
WRETMAN, JH
DOI：
10.1016/s0197-2456(01)00201-x
发表时间：
2002-04-01
期刊：
CONTROLLED CLINICAL TRIALS
影响因子：
--
作者：
Lesaffre, E;Bogaerts, K;Bluhmki, E
通讯作者：
Bluhmki, E
DOI：
10.1111/1467-9884.00227
发表时间：
2000-01-01
期刊：
JOURNAL OF THE ROYAL STATISTICAL SOCIETY SERIES D-THE STATISTICIAN
影响因子：
--
作者：
Senn, S
通讯作者：
Senn, S
影响因子：
1.8
作者：
Stefanski, LA;Boos, DD
通讯作者：
Boos, DD