将非小细胞肺癌患者的健康状态效用建模为时间转化到死亡的过程。
Modelling Health State Utilities as a Transformation of Time to Death in Patients with Non-Small Cell Lung Cancer.
发表日期:2023 Sep 14
作者:
Anthony J Hatswell, Mohammad A Chaudhary, Giles Monnickendam, Alejandro Moreno-Koehler, Katie Frampton, James W Shaw, John R Penrod, Rachael Lawrance
来源:
Cell Death & Disease
摘要:
当使用时间至死(TTD)分析效用时,历来是通过“分组”的方式将观测结果划分为离散的时间段,以创建健康状态效用。我们扩展了这种方法,使用连续函数避免了对分组的假设。所得模型用于测试不同地区和不同国家的数据。我们使用晚期非小细胞肺癌(NSCLC)的五年随访数据,利用广义估计方程拟合了六个连续TTD模型,并将其与基于疾病进展的效用和先前发表的TTD分组进行比较。我们对仅使用有确认死亡的患者数据,仅使用生命的最后一年数据以及人为将数据截断在24个月进行了敏感性分析。然后,将统计上最适配的模型应用于不同地区和不同EQ-5D-3L国家定价模型的数据子集中。[Formula: see text]和[Formula: see text]连续模型的表现优于其他连续模型、分组TTD和基于疾病进展的模型(平均绝对误差和准信息准则方面)。这一结果在敏感性和情景分析中保持一致。患者临近死亡时效用下降的模式在地区和使用首选模型[Formula: see text]的EQ-5D定价模型中是一致的。使用连续模型提供了比TTD分组更好的统计拟合,并且不需要对患者经历的健康状态做出强假设。在需要使用TTD方法进行建模时,应考虑使用连续函数,同时扩大测试候选模型数量和治疗领域的范围,以进一步提高统计拟合度。© 2023年。作者们。
When utilities are analyzed by time to death (TTD), this has historically been implemented by 'grouping' observations as discrete time periods to create health state utilities. We extended the approach to use continuous functions, avoiding assumptions around groupings. The resulting models were used to test the concept with data from different regions and different country tariffs.Five-year follow-up in advanced non-small cell lung cancer (NSCLC) was used to fit six continuous TTD models using generalized estimating equations, which were compared with progression-based utilities and previously published TTD groupings. Sensitivity analyses were performed using only patients with a confirmed death, the last year of life only, and artificially censoring data at 24 months. The statistically best-fitting model was then applied to data subsets by region and different EQ-5D-3L country tariffs.Continuous (natural) [Formula: see text] and [Formula: see text] models outperformed other continuous models, grouped TTD, and progression-based models in statistical fit (mean absolute error and Quasi Information Criterion). This held through sensitivity and scenario analyses. The pattern of reduced utility as a patient approaches death was consistent across regions and EQ-5D tariffs using the preferred [Formula: see text] model.The use of continuous models provides a statistically better fit than TTD groupings, without the need for strong assumptions about the health states experienced by patients. Where a TTD approach is merited for use in modelling, continuous functions should be considered, with the scope for further improvements in statistical fit by both widening the number of candidate models tested and the therapeutic areas investigated.© 2023. The Author(s).