Molinaro Annette M, Wrensch Margaret R, Jenkins Robert B, Eckel-Passow Jeanette E
Department of Neurological Surgery, University of California San Francisco (UCSF), San Francisco, California (A.M.M., M.R.W.); Department of Epidemiology and Biostatistics, University of California San Francisco, San Francisco, California (A.M.M., M.R.W.); Institute of Human Genetics, University of California San Francisco, San Francisco, California (M.R.W.); Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota (R.B.J.); Division of Biomedical Statistics and Informatics, Mayo Clinic, Rochester, Minnesota (J.E.E.-P.).
Neuro Oncol. 2016 May;18(5):609-23. doi: 10.1093/neuonc/nov255. Epub 2015 Dec 8.
Given the lack of beneficial treatments in glioma, there is a need for prognostic models for therapeutic decision making and life planning. Recently several studies defining subtypes of glioma have been published. Here, we review the statistical considerations of how to build and validate prognostic models, explain the models presented in the current glioma literature, and discuss advantages and disadvantages of each model. The 3 statistical considerations to establishing clinically useful prognostic models are: study design, model building, and validation. Careful study design helps to ensure that the model is unbiased and generalizable to the population of interest. During model building, a discovery cohort of patients can be used to choose variables, construct models, and estimate prediction performance via internal validation. Via external validation, an independent dataset can assess how well the model performs. It is imperative that published models properly detail the study design and methods for both model building and validation. This provides readers the information necessary to assess the bias in a study, compare other published models, and determine the model's clinical usefulness. As editors, reviewers, and readers of the relevant literature, we should be cognizant of the needed statistical considerations and insist on their use.
鉴于胶质瘤缺乏有效的治疗方法,需要用于治疗决策和生活规划的预后模型。最近发表了几项定义胶质瘤亚型的研究。在此,我们回顾构建和验证预后模型的统计学考量,解释当前胶质瘤文献中提出的模型,并讨论每个模型的优缺点。建立具有临床实用性的预后模型的三个统计学考量是:研究设计、模型构建和验证。精心的研究设计有助于确保模型无偏倚且可推广到感兴趣的人群。在模型构建过程中,患者的发现队列可用于选择变量、构建模型并通过内部验证估计预测性能。通过外部验证,独立数据集可以评估模型的表现。已发表的模型必须适当地详细说明研究设计以及模型构建和验证的方法。这为读者提供了评估研究偏差、比较其他已发表模型以及确定模型临床实用性所需的信息。作为相关文献的编辑、审稿人和读者,我们应该认识到所需的统计学考量并坚持使用它们。