Model-based effectiveness and cost-effectiveness of risk-based selection strategies for adjuvant chemotherapy in Dutch stage II colon cancer patients

Gabrielle Jongeneel; Marjolein J E Greuter; Felice N van Erning; Miriam Koopman; Geraldine R Vink; Cornelis J A Punt; Veerle M H Coupé

doi:10.1177/1756284821995715

Model-based effectiveness and cost-effectiveness of risk-based selection strategies for adjuvant chemotherapy in Dutch stage II colon cancer patients

Therap Adv Gastroenterol. 2021 Mar 12:14:1756284821995715. doi: 10.1177/1756284821995715. eCollection 2021.

Authors

Gabrielle Jongeneel¹, Marjolein J E Greuter², Felice N van Erning³, Miriam Koopman⁴, Geraldine R Vink³, Cornelis J A Punt⁵, Veerle M H Coupé²

Affiliations

¹ Department of Epidemiology and Data Science, Amsterdam UMC, VU University, PO Box 7057, MF F-wing, Amsterdam, 1007 MB, the Netherlands.
² Department of Epidemiology and Data Science, Amsterdam UMC, VU University, Amsterdam, The Netherlands.
³ Department of Research and Development, Netherlands Comprehensive Cancer Organisation (IKNL), Utrecht, The Netherlands.
⁴ Department of medical oncology, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
⁵ Department of Epidemiology, University Medical Center Utrecht, Julius Center for Health Sciences, Utrecht, The Netherlands.

Abstract

Background: 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.

Methods: 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 MSS status combined with a pT4 stage or a mutation in BRAF and/or KRAS receive chemotherapy assuming perfect adherence and (5) biomarker mutation AND pT4 stage strategy in which patients with MSS status combined with a pT4 stage tumor and a BRAF and/or KRAS 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.

Results: 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.

Conclusion: 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.

Keywords: adjuvant chemotherapy; biomarkers; colon cancer; cost-effectiveness; personalized medicine; stage II.