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Estimating adjuvant treatment effects in Stage II colon cancer: Comparing the synthesis of randomized clinical trial data to real-world data
Authors:Gabrielle Jongeneel  Thomas Klausch  Felice N. van Erning  Geraldine R. Vink  Miriam Koopman  Cornelis J.A. Punt  Marjolein J.E. Greuter  Veerle M.H. Coupé
Affiliation:1. Department of Epidemiology and Biostatistics, Amsterdam UMC, VU University, Amsterdam, The Netherlands;2. Department of Research, Netherlands Comprehensive Cancer Organisation (IKNL), Utrecht, The Netherlands;3. Department of Research, Netherlands Comprehensive Cancer Organisation (IKNL), Utrecht, The Netherlands

University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands;4. University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands;5. Department of medical oncology, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands

Abstract:
There is an ongoing discussion regarding the impact of adjuvant chemotherapy in Stage II colon cancer. We therefore estimated adjuvant treatment effect in Stage II colon cancer using pooled disease-free survival (DFS) data from randomized clinical trials (RCT approach) and compared this to real-world data (RWD approach) estimates. First, we estimated the treatment effect in RCTs by (i) searching relevant trials reporting DFS data, (ii) generating patient-level data from reported DFS data and (iii) estimating treatment effect in the patient-level data. Second, the treatment effect was estimated in an observational cohort of 1,947 patients provided by the Netherlands Cancer Registry using three propensity score methods; matching, weighting and stratification. In the RCT approach, patient-level data of 4,489 patients (events: 853) were generated from seven trials which compared two of the following treatment arms: control, 5FU/LV or FOLFOX. A Cox model was used to estimate a hazard ratio (HR) of 0.77 (0.43;1.10) for 5FU/LV vs. control and 0.93 (0.72;1.15) for FOLFOX vs. 5FU/LV. In the RWD approach, HRs for any adjuvant treatment vs. control were 0.95 (0.50;1.80), 0.88 (0.24;3.21) and 1.05 (0.04;2.06) using matching, weighting and stratification, respectively. There was no significant difference with the estimates from the RCT approach (interaction test, p > 0.10). The RCT data suggest a clinically relevant benefit of adjuvant chemotherapy in terms of DFS, but the estimate did not reach statistical significance. Stratified analyses are required to evaluate whether treatment effect differs in specific subgroups.
Keywords:colon cancer  treatment effect  randomized clinical trial  real-world data  adjuvant chemotherapy
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