Efficacy-Effectiveness Analysis of Encorafenib-Cetuximab Treatment in Patients with Oncogene-Mutated Metastatic Colorectal Cancer: A Cohort Study

Research Background

With the advancement of personalized medicine, targeted therapy has become an important means to treat various cancers. In metastatic colorectal cancer (mCRC), the combination of encorafenib and cetuximab is considered effective for pre-treated BRAF^V600E^ mutant patients. However, even treatments proven effective in randomized controlled clinical trials (such as the BEACON trial) may result in efficacy-effectiveness gaps due to varying real-world application conditions. Given that retrospective cohort studies can provide a more comprehensive understanding of the general patient population, this study aims to assess the real-world effectiveness of this drug combination treatment in clinical practice.

Study Sources

• Authors: Koen Zwart, Sietske C. M. W. van Nassau, Frederieke H. Van der Baan, Miriam Koopman, Petur Snaebjornsson, Anna J. Van Gestel, Geraldine R. Vink, Jeanine M. L. Roodhart
• Institutions: University Medical Center Utrecht, Amsterdam Cancer Institute, Netherlands Comprehensive Cancer Organisation (IKNL)
• Publication Date: 2024
• Journal: British Journal of Cancer

Study Details

• Subjects: 166 Dutch mCRC patients treated with encorafenib-cetuximab.
• Methods: Collected pathology reports and individual case data, and obtained gene mutation analysis results by correlating with the Dutch Nationwide Pathology Database (PALGA).
• Results: The study observed a median overall survival (OS) of 6.7 months in practical use, significantly differing from the 9.3 months reported in the BEACON trial. Among them, 36% of patients were deemed ineligible for inclusion in the BEACON trial, and this subgroup had a generally shorter OS.
• Conclusion: In actual clinical application, the efficacy of this treatment differs from the results obtained in clinical trials, particularly for patients with significant brain metastasis symptoms and/or WHO Quality of Life Score ≥2, where the treatment effect is poor.

Significance and Highlights of the Study

This study emphasizes the need to focus on the discrepancies between real-world data and clinical trial results, underscoring the value of considering patient groups that do not meet the inclusion criteria of clinical trials during data analysis. The study provides a comprehensive understanding of the metastatic colorectal cancer patient population, reinforcing the necessity of personalized treatment recommendations in actual clinical settings.

Additional Information

• The study did not involve adverse reaction data nor set up a control group, limiting comprehensive assessment of the treatment benefits and drawbacks.
• We need to combine the efficacy data from randomized controlled trials with real-world study results to offer evidence-based recommendations in treatment guidelines.