Cooner Freda, Banerjee Sudipto, Carlin Bradley P, Sinha Debajyoti
Division of Biostatistics, Office of Surveillance and Biometrics, Center for Devices and Radiological Health, Food and Drug Administration.
J Am Stat Assoc. 2007 Jun 1;102(478):560-572. doi: 10.1198/016214507000000112.
With rapid improvements in medical treatment and health care, many datasets dealing with time to relapse or death now reveal a substantial portion of patients who are cured (i.e., who never experience the event). Extended survival models called cure rate models account for the probability of a subject being cured and can be broadly classified into the classical mixture models of Berkson and Gage (BG type) or the stochastic tumor models pioneered by Yakovlev and extended to a hierarchical framework by Chen, Ibrahim, and Sinha (YCIS type). Recent developments in Bayesian hierarchical cure models have evoked significant interest regarding relationships and preferences between these two classes of models. Our present work proposes a unifying class of cure rate models that facilitates flexible hierarchical model-building while including both existing cure model classes as special cases. This unifying class enables robust modeling by accounting for uncertainty in underlying mechanisms leading to cure. Issues such as regressing on the cure fraction and propriety of the associated posterior distributions under different modeling assumptions are also discussed. Finally, we offer a simulation study and also illustrate with two datasets (on melanoma and breast cancer) that reveal our framework's ability to distinguish among underlying mechanisms that lead to relapse and cure.
随着医疗和卫生保健的迅速改善,许多涉及复发时间或死亡时间的数据集现在显示,有很大一部分患者被治愈(即从未经历该事件)。称为治愈率模型的扩展生存模型考虑了受试者被治愈的概率,并且可以大致分为Berkson和Gage的经典混合模型(BG类型)或由Yakovlev开创并由Chen、Ibrahim和Sinha扩展到分层框架的随机肿瘤模型(YCIS类型)。贝叶斯分层治愈率模型的最新发展引起了人们对这两类模型之间关系和偏好的极大兴趣。我们目前的工作提出了一类统一的治愈率模型,该模型有助于灵活地构建分层模型,同时将现有的治愈率模型类作为特殊情况包含在内。这一统一的类别通过考虑导致治愈的潜在机制中的不确定性来实现稳健建模。还讨论了诸如治愈率回归以及在不同建模假设下相关后验分布的恰当性等问题。最后,我们进行了一项模拟研究,并通过两个数据集(关于黑色素瘤和乳腺癌)进行说明,这些数据集揭示了我们的框架区分导致复发和治愈的潜在机制的能力。