Gail Mitchell H, Pfeiffer Ruth M
Biostatistics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Executive Plaza South, EPS 8032, Bethesda, MD 20892-7244, USA.
Biostatistics. 2005 Apr;6(2):227-39. doi: 10.1093/biostatistics/kxi005.
Absolute risk is the probability that an individual who is free of a given disease at an initial age, a, will develop that disease in the subsequent interval (a, t]. Absolute risk is reduced by mortality from competing risks. Models of absolute risk that depend on covariates have been used to design intervention studies, to counsel patients regarding their risks of disease and to inform clinical decisions, such as whether or not to take tamoxifen to prevent breast cancer. Several general criteria have been used to evaluate models of absolute risk, including how well the model predicts the observed numbers of events in subsets of the population ("calibration"), and "discriminatory power," measured by the concordance statistic. In this paper we review some general criteria and develop specific loss function-based criteria for two applications, namely whether or not to screen a population to select subjects for further evaluation or treatment and whether or not to use a preventive intervention that has both beneficial and adverse effects. We find that high discriminatory power is much more crucial in the screening application than in the preventive intervention application. These examples indicate that the usefulness of a general criterion such as concordance depends on the application, and that using specific loss functions can lead to more appropriate assessments.
绝对风险是指在初始年龄a时未患特定疾病的个体在随后时间段(a, t]内患该疾病的概率。绝对风险会因竞争风险导致的死亡率而降低。依赖协变量的绝对风险模型已被用于设计干预研究、为患者提供疾病风险咨询以及为临床决策提供依据,比如是否服用他莫昔芬来预防乳腺癌。已有若干通用标准被用于评估绝对风险模型,包括模型对人群子集中观察到的事件数量的预测能力(“校准”),以及通过一致性统计量衡量的“区分能力”。在本文中,我们回顾了一些通用标准,并针对两种应用开发了基于特定损失函数的标准,这两种应用分别是是否对人群进行筛查以选择进一步评估或治疗的对象,以及是否使用具有利弊兼具的预防性干预措施。我们发现,高区分能力在筛查应用中比在预防性干预应用中更为关键。这些例子表明,诸如一致性这样的通用标准的有用性取决于具体应用,而使用特定损失函数能够得出更恰当的评估。