基于含治愈患者生存数据的Cox-TEL方法性能的模拟研究  

A simulation study of the reliability and accuracy of Cox-TEL method for estimating hazard ratio and difference in proportions for long-term survival data containing cured patients

作　　者：邹碧清 徐利珊 李柯柯 安胜利 ZOU Biqing;XU Lishan;LI Keke;AN Shengi(Department of Biostatistics,School of Public Health,Southern Medical University,Guangzhou 510515,China)

机构地区：[1]南方医科大学公共卫生学院生物统计学系,广东广州510515

出　　处：《南方医科大学学报》2024年第6期1182-1187,共6页Journal of Southern Medical University

基　　金：广东省自然科学基金(2022A1515012152);南方医科大学2022年度大学生创新创业训练计划项目(S202212121135)。

摘　　要：目的 探究针对长期生存数据的Cox比例风险模型的Taylor拓展调整(Cox-TEL)方法在含治愈患者的生存数据下的适用条件与适用范围。方法 基于Weibull分布模拟生存数据方法,模拟生成不同治愈率、删失率和治愈率差值的生存数据。然后基于Cox-TEL方法进行分析,获得Ⅰ类错误和检验效能对其表现性能进行评价。结果 针对模型的未治愈部分,Cox-TEL方法的Ⅰ类错误略高于0.05,其检验效能在样本量较大、治愈率差值较大时较为理想。针对治愈部分,Cox-TEL方法的Ⅰ类错误能够较好的控制在0.05左右,且在大多数情况下都能保持较高的检验效能。结论 Cox-TEL方法在多数情况下,比如样本量较大、删失率较低、组间治愈率差值较大时,能有效分析未治愈患者数据,得到较可靠的风险比HR值;在不同的样本量、删失率和治愈率下,该方法都能较准确地估计出治愈患者两组治愈率差值。Objective To explore the applicable conditions of the Cox-TEL(Cox PH-Taylor expansion adjustment for long-term survival data)method for analysis of survival data that contain cured patients.Methods The simulated survival data method based on Weibull distribution was used to simulate and generate the survival data with different cure rates,censored rates,and cure rate differences.The Cox-TEL method was used for analysis of the generated simulation data,and its performance was evaluated by calculating its type I error and power.Results Almost all the type I error of the hazard ratios(HRs)obtained by the Cox-TEL method under different conditions were slightly greater than 0.05,and this method showed a good test power for estimating the HRs for data with a large sample size and a large difference in proportions(DPs).For the data of cured patients,the type I error of the DPs obtained by the Cox-TEL method was well around 0.05,and its test power was robust in most of the scenarios.Conclusion The Cox-TEL method is effective for analyzing data of uncured patients and obtaining reliable HRs for most of the survival data with a sample size,a low censored rates,and a large difference in cure rates.The method is capable of accurately estimating the DPs regardless of the sample size,censored rates,or the cure rates.

关 键 词：Cox-TEL 治愈模型 生存分析 检验效能 

分 类 号：R195.1[医药卫生—卫生统计学]