Department of Epidemiology and Biostatistics, Amsterdam UMC, VU University, MF F-wing, PO Box 7057, 1007 MB, Amsterdam, The Netherlands.
Department of Research, Netherlands Comprehensive Cancer Organization (IKNL), Utrecht, The Netherlands.
Eur J Health Econ. 2020 Sep;21(7):1059-1073. doi: 10.1007/s10198-020-01199-4. Epub 2020 May 26.
To develop a decision model for the population-level evaluation of strategies to improve the selection of stage II colon cancer (CC) patients who benefit from adjuvant chemotherapy.
A Markov cohort model with a one-month cycle length and a lifelong time horizon was developed. Five health states were included; diagnosis, 90-day mortality, death other causes, recurrence and CC death. Data from the Netherlands Cancer Registry were used to parameterize the model. Transition probabilities were estimated using parametric survival models including relevant clinical and pathological covariates. Subsequently, biomarker status was implemented using external data. Treatment effect was incorporated using pooled trial data. Model development, data sources used, parameter estimation, and internal and external validation are described in detail. To illustrate the use of the model, three example strategies were evaluated in which allocation of treatment was based on (A) 100% adherence to the Dutch guidelines, (B) observed adherence to guideline recommendations and (C) a biomarker-driven strategy.
Overall, the model showed good internal and external validity. Age, tumor growth, tumor sidedness, evaluated lymph nodes, and biomarker status were included as covariates. For the example strategies, the model predicted 83, 87 and 77 CC deaths after 5 years in a cohort of 1000 patients for strategies A, B and C, respectively.
This model can be used to evaluate strategies for the allocation of adjuvant chemotherapy in stage II CC patients. In future studies, the model will be used to estimate population-level long-term health gain and cost-effectiveness of biomarker-based selection strategies.
开发一种用于评估改善受益于辅助化疗的 II 期结肠癌（CC）患者选择策略的人群水平的决策模型。
建立了一个具有一个月周期长度和终身时间范围的马尔可夫队列模型。纳入了五个健康状态：诊断、90 天死亡率、其他原因死亡、复发和 CC 死亡。使用荷兰癌症登记处的数据对模型进行参数化。使用包括相关临床和病理协变量的参数生存模型估计转移概率。随后，使用外部数据实施生物标志物状态。使用汇总试验数据纳入治疗效果。详细描述了模型开发、使用的数据来源、参数估计以及内部和外部验证。为了说明模型的使用，评估了三种示例策略，其中治疗的分配基于（A）100%遵循荷兰指南，（B）观察到的对指南建议的依从性和（C）基于生物标志物的策略。
总体而言，该模型显示出良好的内部和外部有效性。年龄、肿瘤生长、肿瘤侧别、评估的淋巴结和生物标志物状态被纳入协变量。对于示例策略，模型预测在 1000 名患者队列中，策略 A、B 和 C 分别在 5 年后有 83、87 和 77 例 CC 死亡。
该模型可用于评估 II 期 CC 患者辅助化疗分配的策略。在未来的研究中，该模型将用于估计基于生物标志物选择策略的人群水平的长期健康获益和成本效益。
