应用线性混合模型估计重复测量的临床试验数据的可靠性

Tony Vangeneugden , Annouschka Laenen , Helena Geys , Didier Renard , Geert Molenberghs
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引用次数: 71

摘要

重复测量被用来研究精神健康科学背景下的可靠性。说明了当量表为连续或准连续时,如何使用线性混合模型推导测重信度。这种方法的优点是可以充分利用混合模型的建模能力。具有不同平均结构的重复测量可用于有效地研究可靠性,校正协变量效应是可能的,并且允许测量之间存在复杂的方差-协方差结构。当方差结构降为随机截距(复合对称)时,恢复经典方法。对于更复杂的方差结构(例如,包括时间和/或序列相关的随机斜率),可以获得时变可靠度函数。该方法的动机是应用于五项双盲随机临床试验的数据,这些试验比较了利培酮与传统抗精神病药物治疗慢性精神分裂症的效果。通过残差图和调查有影响的观测值的影响来调查模型假设。
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Applying linear mixed models to estimate reliability in clinical trial data with repeated measurements

Repeated measures are exploited to study reliability in the context of psychiatric health sciences. It is shown how test–retest reliability can be derived using linear mixed models when the scale is continuous or quasi-continuous. The advantage of this approach is that the full modeling power of mixed models can be used. Repeated measures with a different mean structure can be used to usefully study reliability, correction for covariate effects is possible, and a complicated variance–covariance structure between measurements is allowed. In case the variance structure reduces to a random intercept (compound symmetry), classical methods are recovered. With more complex variance structures (e.g., including random slopes of time and/or serial correlation), time-dependent reliability functions are obtained. The methodology is motivated by and applied to data from five double-blind randomized clinical trials comparing the effects of risperidone to conventional antipsychotic agents for the treatment of chronic schizophrenia. Model assumptions are investigated through residual plots and by investigating the effect of influential observations.

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