On estimating the time to statistical cure
On estimating the time to statistical cure
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DOI：
10.1186/s12874-020-00946-8
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发表时间：
2020-03-26
影响因子：
4
通讯作者：
Bogsted, Martin
中科院分区：
文献类型：
--
作者：
Jakobsen, Lasse H.;Andersson, Therese M. -L.;Bogsted, Martin
背景 与一般人群相比，从诊断之时起测量的癌症患者的死亡风险通常较高。然而，对于某些癌症类型，随着时间的推移，患者死亡风险将达到与一般人群死亡风险相同的水平。死亡风险达到与一般人群相同水平的时间点称为治愈点，患者、临床医生和医疗保健规划者对此非常感兴趣。在以前的研究中，治愈点的估计是以临时方式处理的，通常没有考虑临床相关性的边际。方法 我们回顾了现有的估计治愈点的方法，并讨论了量化癌症患者与普通人群之间死亡率差异的新的临床相关措施，这些措施可用于治愈点估计。这些方法的性能在模拟研究中进行了评估，并根据丹麦结肠癌患者的生存数据说明了这些方法。结果模拟显示，估计治愈点的偏差取决于为量化超额死亡率而选择的措施、所选择的临床相关性裕度以及所应用的估计程序。这些选择是相互依赖的，因为死亡率测量值的选择既取决于定义临床相关性范围的能力，也取决于准确计算死亡率测量值的能力。癌症生存数据的分析证明了考虑估计治愈点的置信区间的重要性，因为在某些情况下这些置信区间可能很宽，限制了估计治愈点的适用性。结论 尽管治愈点在临床上很有吸引力并且具有广泛的适用性，但估计仍然是一项艰巨的任务。该估计依赖于许多选择，每个选择都与从业者应该意识到的陷阱相关。
Background The mortality risk among cancer patients measured from the time of diagnosis is often elevated in comparison to the general population. However, for some cancer types, the patient mortality risk will over time reach the same level as the general population mortality risk. The time point at which the mortality risk reaches the same level as the general population is called the cure point and is of great interest to patients, clinicians, and health care planners. In previous studies, estimation of the cure point has been handled in an ad hoc fashion, often without considerations about margins of clinical relevance. Methods We review existing methods for estimating the cure point and discuss new clinically relevant measures for quantifying the mortality difference between cancer patients and the general population, which can be used for cure point estimation. The performance of the methods is assessed in a simulation study and the methods are illustrated on survival data from Danish colon cancer patients. Results The simulations revealed that the bias of the estimated cure point depends on the measure chosen for quantifying the excess mortality, the chosen margin of clinical relevance, and the applied estimation procedure. These choices are interdependent as the choice of mortality measure depends both on the ability to define a margin of clinical relevance and the ability to accurately compute the mortality measure. The analysis of cancer survival data demonstrates the importance of considering the confidence interval of the estimated cure point, as these may be wide in some scenarios limiting the applicability of the estimated cure point. Conclusions Although cure points are appealing in a clinical context and has widespread applicability, estimation remains a difficult task. The estimation relies on a number of choices, each associated with pitfalls that the practitioner should be aware of.