Department of Medical Epidemiology and Biostatistics, Karolinska Institutet, Box 281, 171 77 Stockholm, Sweden.
BMC Med Res Methodol. 2011 Jun 22;11:96. doi: 10.1186/1471-2288-11-96.
When the mortality among a cancer patient group returns to the same level as in the general population, that is, the patients no longer experience excess mortality, the patients still alive are considered "statistically cured". Cure models can be used to estimate the cure proportion as well as the survival function of the "uncured". One limitation of parametric cure models is that the functional form of the survival of the "uncured" has to be specified. It can sometimes be hard to find a survival function flexible enough to fit the observed data, for example, when there is high excess hazard within a few months from diagnosis, which is common among older age groups. This has led to the exclusion of older age groups in population-based cancer studies using cure models.
Here we have extended the flexible parametric survival model to incorporate cure as a special case to estimate the cure proportion and the survival of the "uncured". Flexible parametric survival models use splines to model the underlying hazard function, and therefore no parametric distribution has to be specified.
We have compared the fit from standard cure models to our flexible cure model, using data on colon cancer patients in Finland. This new method gives similar results to a standard cure model, when it is reliable, and better fit when the standard cure model gives biased estimates.
Cure models within the framework of flexible parametric models enables cure modelling when standard models give biased estimates. These flexible cure models enable inclusion of older age groups and can give stage-specific estimates, which is not always possible from parametric cure models.
当癌症患者群体的死亡率恢复到与普通人群相同的水平时,即患者不再经历超额死亡率,那么仍存活的患者被认为是“统计学治愈”。治愈模型可用于估计治愈比例以及“未治愈”人群的生存函数。参数治愈模型的一个局限性是必须指定“未治愈”人群的生存函数的形式。有时,很难找到一个足够灵活的生存函数来拟合观察到的数据,例如,在诊断后几个月内存在高超额风险,这种情况在老年人群中很常见。这导致在使用治愈模型的基于人群的癌症研究中排除了老年人群。
在这里,我们将灵活的参数生存模型扩展为包含治愈作为特殊情况,以估计治愈比例和“未治愈”人群的生存。灵活的参数生存模型使用样条来对潜在的风险函数进行建模,因此不必指定参数分布。
我们使用芬兰结肠癌患者的数据比较了标准治愈模型和我们的灵活治愈模型的拟合效果。当标准治愈模型可靠时,这种新方法与标准治愈模型的拟合结果相似,而当标准治愈模型给出有偏差的估计时,它的拟合效果更好。
在灵活参数模型框架内的治愈模型可以在标准模型给出有偏差的估计时进行治愈建模。这些灵活的治愈模型可以纳入老年人群,并可以提供特定于分期的估计,这在参数治愈模型中并不总是可行的。