Jongeneel Gabrielle, Greuter Marjolein J E, van Erning Felice N, Koopman Miriam, Vink Geraldine R, Punt Cornelis J A, Coupé Veerle M H
Department of Epidemiology and Data Science, Amsterdam UMC, VU University, PO Box 7057, MF F-wing, Amsterdam, 1007 MB, the Netherlands.
Department of Epidemiology and Data Science, Amsterdam UMC, VU University, Amsterdam, The Netherlands.
Therap Adv Gastroenterol. 2021 Mar 12;14:1756284821995715. doi: 10.1177/1756284821995715. eCollection 2021.
We aimed to evaluate the cost-effectiveness of risk-based strategies to improve the selection of surgically treated stage II colon cancer (CC) patients for adjuvant chemotherapy.
Using the 'Personalized Adjuvant TreaTment in EaRly stage coloN cancer' (PATTERN) model, we evaluated five selection strategies: (1) no chemotherapy, (2) Dutch guideline recommendations assuming observed adherence, (3) Dutch guideline recommendations assuming perfect adherence, (4) biomarker mutation OR pT4 stage strategy in which patients with status combined with a pT4 stage or a mutation in and/or receive chemotherapy assuming perfect adherence and (5) biomarker mutation AND pT4 stage strategy in which patients with status combined with a pT4 stage tumor and a and/or mutation receive chemotherapy assuming perfect adherence. Outcomes were number of CC deaths per 1000 patients and total discounted costs and quality-adjusted life-years (QALYs) per patient (pp). Analyses were conducted from a societal perspective. The robustness of model predictions was assessed in sensitivity analyses.
The reference strategy, that is, no adjuvant chemotherapy, resulted in 139 CC deaths in a cohort of 1000 patients, 8.077 QALYs pp and total costs of €22,032 pp. Strategies 2-5 were more effective (range 8.094-8.217 QALYs pp and range 118-136 CC deaths per 1000 patients) and more costly (range €22,404-€25,102 pp). Given a threshold of €50,000/QALY, the optimal use of resources would be to treat patients with either the full adherence strategy and biomarker mutation OR pT4 stage strategy.
Selection of stage II CC patients for chemotherapy can be improved by either including biomarker status in the selection strategy or by improving adherence to the Dutch guideline recommendations.
我们旨在评估基于风险的策略在改善手术治疗的II期结肠癌（CC）患者辅助化疗选择方面的成本效益。
使用“早期结肠癌个性化辅助治疗”（PATTERN）模型，我们评估了五种选择策略：（1）不进行化疗；（2）假设观察到的依从性的荷兰指南建议；（3）假设完全依从性的荷兰指南建议；（4）生物标志物突变或pT4期策略，即具有特定状态且合并pT4期或特定基因和/或其他基因发生突变的患者假设完全依从性接受化疗；（5）生物标志物突变且pT4期策略，即具有特定状态且合并pT4期肿瘤以及特定基因和/或其他基因发生突变的患者假设完全依从性接受化疗。结果指标为每1000例患者的CC死亡人数以及每位患者（pp）的总贴现成本和质量调整生命年（QALY）。分析从社会角度进行。在敏感性分析中评估模型预测的稳健性。
参考策略，即不进行辅助化疗，在1000例患者队列中导致139例CC死亡，每位患者8.077个QALY，总费用为每位患者22,032欧元。策略2 - 5更有效（范围为每位患者8.094 - 8.217个QALY，每1000例患者118 - 136例CC死亡）且成本更高（范围为每位患者22,404 - 25,102欧元）。给定每QALY 50,000欧元的阈值，资源的最佳利用方式是采用完全依从性策略以及生物标志物突变或pT4期策略来治疗患者。
通过在选择策略中纳入生物标志物状态或提高对荷兰指南建议的依从性，可以改善II期CC患者化疗的选择。
