Real-world effectiveness of regorafenib in the treatment of patients with metastatic colorectal cancer: A retrospective, observational study

Asia Pac J Clin Oncol. 2023 Oct;19(5):e291-e299. doi: 10.1111/ajco.13909. Epub 2022 Dec 26.

Abstract

Aim: To evaluate the real-world usage pattern and factors associated with the effectiveness of regorafenib in patients with metastatic colorectal cancer (mCRC).

Methods: This retrospective study analyzed data for 209 patients with mCRC treated with regorafenib as third or later line of therapy. TheKaplan-Meier method was used to draw the survival curves. Cox proportional hazard regression models were used to analyze the prognostic value for overall survival (OS).

Results: Of 209 patients, 156 (75%) were treated with regorafenib, and 53 (25%) were given regorafenib combined with programmed cell death-1 (PD-1) inhibitors. About 182 (87%) patients had a dose record of regorafenib. The initial daily doses of regorafenib were 160, 120, 80, and 40 mg, accounting for 29%, 17%, 48%, and 6% of patients, respectively. The median follow-up time was 11.3 months, and the median OS was 12.0 months (95% CI: 9.7-14.3). Patients treated with PD-1 inhibitors plus regorafenib had a longer OS than the non-PD-1 group (13.5 vs. 10.1 months, hazard ratio [HR] = .534, 95% CI: .325-.879; p = .014). A total of 49 patients with microsatellite stable or mismatch repair-proficient genotype treated with PD-1 inhibitors plus regorafenib had a longer OS than the non-PD-1 group (13.5 vs. 9.7 months; HR = .563, p = .027). The median OS of patients continuing treatment with regorafenib after progression (n = 19, with five patients receiving additional immunotherapy) was marginally longer than patients discontinuing regorafenib after progression (12.7 vs. 11.9 months, p = .425) observed in a smaller cohort.

Conclusion: In real-world practice, patients with mCRC in whom standard regimens had failed have a good survival benefit with regorafenib. Combination with PD-1 inhibitor may further prolong survival of the patients.

Keywords: metastatic colorectal cancer; real-world; regorafenib; survival analysis.

Publication types

  • Observational Study

MeSH terms

  • Colonic Neoplasms* / drug therapy
  • Colorectal Neoplasms* / pathology
  • Humans
  • Immune Checkpoint Inhibitors / therapeutic use
  • Phenylurea Compounds / therapeutic use
  • Pyridines / therapeutic use
  • Rectal Neoplasms* / drug therapy
  • Retrospective Studies

Substances

  • regorafenib
  • Immune Checkpoint Inhibitors
  • Pyridines
  • Phenylurea Compounds