Sies Aniek, Demyttenaere Koen, Van Mechelen Iven
a Faculty of Psychology and Educational Sciences , KU Leuven , Leuven , Belgium.
b Department of Neurosciences , KU Leuven , Leuven , Belgium.
J Biopharm Stat. 2019;29(3):491-507. doi: 10.1080/10543406.2019.1579220. Epub 2019 Feb 22.
Precision medicine, in the sense of tailoring the choice of medical treatment to patients' pretreatment characteristics, is nowadays gaining a lot of attention. Preferably, this tailoring should be realized in an evidence-based way, with key evidence in this regard pertaining to subgroups of patients that respond differentially to treatment (i.e., to subgroups involved in treatment-subgroup interactions). Often a-priori hypotheses on subgroups involved in treatment-subgroup interactions are lacking or are incomplete at best. Therefore, methods are needed that can induce such subgroups from empirical data on treatment effectiveness in a manner. Recently, quite a few such methods have been developed. So far, however, there is little empirical experience in their usage. This may be problematic for medical statisticians and statistically minded medical researchers, as many (nontrivial) choices have to be made during the data-analytic process. The main purpose of this paper is to discuss the major concepts and considerations when using these methods. This discussion will be based on a systematic, conceptual, and technical analysis of the type of research questions at play, and of the type of data that the methods can handle along with the available software, and a review of available empirical evidence. We will illustrate all this with the analysis of a dataset comparing several anti-depressant treatments.
精准医学，即根据患者的治疗前特征来定制医疗治疗方案的选择，如今正受到广泛关注。理想情况下，这种定制应以循证方式实现，这方面的关键证据涉及对治疗反应不同的患者亚组（即参与治疗-亚组相互作用的亚组）。通常，关于参与治疗-亚组相互作用的亚组的先验假设要么缺乏，要么充其量也不完整。因此，需要能够从治疗效果的经验数据中以某种方式诱导出此类亚组的方法。最近，已经开发了不少这样的方法。然而，到目前为止，它们的使用经验还很少。这对于医学统计学家和有统计学思维的医学研究人员来说可能是个问题，因为在数据分析过程中必须做出许多（重要的）选择。本文的主要目的是讨论使用这些方法时的主要概念和注意事项。这种讨论将基于对所涉及的研究问题类型、这些方法能够处理的数据类型以及可用软件进行系统的、概念性的和技术性的分析，以及对现有经验证据的综述。我们将通过对一个比较几种抗抑郁治疗的数据集的分析来说明这一切。
