Akynkozhayev Birzhan, Christoffersen Benjamin, Liu Xingrong, Humphreys Keith, Clements Mark
Department of Medical Epidemiology and Biostatistics, Karolinska Institutet, Stockholm, Sweden.
Department of Oncology-Pathology, Karolinska Institutet, Stockholm, Sweden.
Biom J. 2025 Oct;67(5):e70074. doi: 10.1002/bimj.70074.
Accelerated failure time (AFT) models offer an attractive alternative to Cox proportional hazards models. AFT models are collapsible and, unlike hazard ratios in proportional hazards models, the acceleration factor-a key effect measure in AFT models-is collapsible, meaning its value remains unchanged when adjusting for additional covariates. In addition, AFT models provide an intuitive interpretation directly on the survival time scale. From the recent development of smooth parametric AFT models, we identify potential issues with their applications and note several desired extensions that have not yet been implemented. To enrich this tool and its application in clinical research, we improve the AFT models within a flexible parametric framework in several ways: we adopt monotone natural splines to constrain the log cumulative hazard to be a monotonic function across its support; allow for time-varying acceleration factors, possibly include cure and accommodating more than one time-varying effect; and implement both mixture and nonmixture cure models. We implement all of these extensions in the rstpm2 package, which is publicly available on CRAN. Simulations highlight a varying success in estimating cure fractions. However, in terms of covariate-effect estimation, flexible AFT models appear to be more robust than the Cox model even when there is a high proportion of cured individuals in the data, regardless of whether cure is reached within the observed data. We also apply some of our extensions of AFT models to real-world survival data.
加速失效时间(AFT)模型为Cox比例风险模型提供了一个有吸引力的替代方案。AFT模型是可折叠的,与比例风险模型中的风险比不同,加速因子(AFT模型中的一个关键效应量度)是可折叠的,这意味着在调整其他协变量时其值保持不变。此外,AFT模型直接在生存时间尺度上提供了直观的解释。从平滑参数AFT模型的最新发展来看,我们识别出其应用中的潜在问题,并指出了几个尚未实现的理想扩展。为了丰富这个工具及其在临床研究中的应用,我们在一个灵活的参数框架内以多种方式改进AFT模型:我们采用单调自然样条来约束对数累积风险在其支持域内为单调函数;允许时变加速因子,可能包括治愈情况并容纳多个时变效应;并实现混合和非混合治愈模型。我们在rstpm2包中实现了所有这些扩展,该包可在CRAN上公开获取。模拟结果突出显示在估计治愈比例方面成功率各不相同。然而,在协变量效应估计方面,即使数据中治愈个体的比例很高,无论在观察数据内是否达到治愈,灵活的AFT模型似乎比Cox模型更稳健。我们还将AFT模型的一些扩展应用于实际生存数据。