带终止事件的特定治疗复发事件均值比例的半参估计  

Semiparametric Estimation of Ratios in Treatment-specific Recurrent Event Means with a Terminal Event

作　　者：马燕 孙晓伟 MA YAN;SUN XIAOWEI(Department of Mathematics,Qilu Normal University,Jinan 250013,China;Department of Mathematics,School of Science,Beijing Jiaotong University,Beijing 100044,China)

机构地区：[1]齐鲁师范学院数学学院,济南250013 [2]北京交通大学理学院数学系,北京100044

出　　处：《应用数学学报》2020年第6期949-965,共17页Acta Mathematicae Applicatae Sinica

摘　　要：复发事件数据在生物医学研究中十分常见,而一个终止事件可能会阻止复发事件的进一步发生.在比较治疗效果的研究中,边际均值常常是人们感兴趣的指标,而且特定治疗事件的均值比例通常不会随着时间推移而保持不变.本文提出了一种半参数方法来比较特定治疗的复发事件均值,该方法结合了针对终止事件的比例风险率模型和针对条件复发事件率的乘性模型.在本文中,治疗效果由特定治疗的复发事件均值比例来衡量.我们给出了该比例的估计过程,并且证明了估计的渐近性质.最后我们通过数值模拟评估了文中所提估计的有限样本性质,并将所提方法应用于膀胱癌数据研究中,从而验证该方法的有效性.Recurrent event data often arise from biomedical studies and a terminal event may preclude further occurrence of recurrent events.In comparing treatments,the marginal mean is frequently of interest,and treatment-specific ratios in the mean number of events are often not constant over time.In this article,we propose a semiparametric method to compare treatment-specific recurrent event means by combining an proportional hazards model for the terminal event and an proportional rates model for the conditional recurrent event rate.The treatment effect is measured by the ratio between treatment-specific recurrent event means.Estimation procedures are developed for the measure and the asymptotic properties of the proposed estimators are established.The finite sample performance of the proposed estimators is examined through simulation studies,and an application to a bladder cancer study demonstrates the usefulness of our method.

关 键 词：乘性模型 边际均值 复发事件 半参数方法 终止事件 治疗效果 

分 类 号：O212.7[理学—概率论与数理统计]