Diaz Francisco J
Department of Biostatistics, The University of Kansas Medical Center, Mail Stop 1026, 3901 Rainbow Blvd., Kansas City, 66160, KS, U.S.A.
Stat Med. 2016 Oct 15;35(23):4077-92. doi: 10.1002/sim.7005. Epub 2016 Jun 20.
We propose statistical definitions of the individual benefit of a medical or behavioral treatment and of the severity of a chronic illness. These definitions are used to develop a graphical method that can be used by statisticians and clinicians in the data analysis of clinical trials from the perspective of personalized medicine. The method focuses on assessing and comparing individual effects of treatments rather than average effects and can be used with continuous and discrete responses, including dichotomous and count responses. The method is based on new developments in generalized linear mixed-effects models, which are introduced in this article. To illustrate, analyses of data from the Sequenced Treatment Alternatives to Relieve Depression clinical trial of sequences of treatments for depression and data from a clinical trial of respiratory treatments are presented. The estimation of individual benefits is also explained. Copyright © 2016 John Wiley & Sons, Ltd.
我们提出了医学或行为治疗个体获益以及慢性病严重程度的统计学定义。这些定义用于开发一种图形方法,统计学家和临床医生可在个性化医疗视角下的临床试验数据分析中使用该方法。该方法侧重于评估和比较治疗的个体效果而非平均效果,可用于连续和离散反应,包括二分反应和计数反应。该方法基于广义线性混合效应模型的新进展,本文对此进行了介绍。为作说明,给出了抑郁症治疗序列的缓解抑郁症的序贯治疗替代方案临床试验数据以及呼吸治疗临床试验数据的分析。还解释了个体获益的估计方法。版权所有© 2016约翰威立父子有限公司。