Asano Junichi, Hirakawa Akihiro, Hamada Chikuma
Biostatistics Group, Center for Product Evaluation, Pharmaceuticals and Medical Devices Agency, Tokyo, 100-0013, Japan.
Pharm Stat. 2014 Nov-Dec;13(6):357-63. doi: 10.1002/pst.1630. Epub 2014 Jul 16.
A cure rate model is a survival model incorporating the cure rate with the assumption that the population contains both uncured and cured individuals. It is a powerful statistical tool for prognostic studies, especially in cancer. The cure rate is important for making treatment decisions in clinical practice. The proportional hazards (PH) cure model can predict the cure rate for each patient. This contains a logistic regression component for the cure rate and a Cox regression component to estimate the hazard for uncured patients. A measure for quantifying the predictive accuracy of the cure rate estimated by the Cox PH cure model is required, as there has been a lack of previous research in this area. We used the Cox PH cure model for the breast cancer data; however, the area under the receiver operating characteristic curve (AUC) could not be estimated because many patients were censored. In this study, we used imputation-based AUCs to assess the predictive accuracy of the cure rate from the PH cure model. We examined the precision of these AUCs using simulation studies. The results demonstrated that the imputation-based AUCs were estimable and their biases were negligibly small in many cases, although ordinary AUC could not be estimated. Additionally, we introduced the bias-correction method of imputation-based AUCs and found that the bias-corrected estimate successfully compensated the overestimation in the simulation studies. We also illustrated the estimation of the imputation-based AUCs using breast cancer data.
治愈率模型是一种生存模型,它纳入了治愈率,并假设总体中既包含未治愈个体也包含已治愈个体。它是预后研究的一种强大统计工具,尤其在癌症研究中。治愈率对于临床实践中的治疗决策很重要。比例风险(PH)治愈率模型可以预测每个患者的治愈率。这包含一个用于治愈率的逻辑回归组件和一个用于估计未治愈患者风险的Cox回归组件。由于此前该领域缺乏研究,因此需要一种用于量化由Cox PH治愈率模型估计的治愈率预测准确性的指标。我们将Cox PH治愈率模型用于乳腺癌数据;然而,由于许多患者被截尾,因此无法估计受试者工作特征曲线(AUC)下的面积。在本研究中,我们使用基于插补的AUC来评估PH治愈率模型中治愈率的预测准确性。我们通过模拟研究检验了这些AUC的精度。结果表明,尽管普通AUC无法估计,但基于插补的AUC在许多情况下是可估计的,并且其偏差小到可以忽略不计。此外,我们引入了基于插补的AUC的偏差校正方法,发现偏差校正估计在模拟研究中成功补偿了高估。我们还使用乳腺癌数据说明了基于插补的AUC的估计。