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

Abstract

AimTo evaluate the real‐world usage pattern and factors associated with the effectiveness of regorafenib in patients with metastatic colorectal cancer (mCRC).MethodsThis 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).ResultsOf 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.ConclusionIn 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.

RNA Expression-Based Analysis to Predict Response in Patients with Metastatic Mismatch Repair Proficient Colorectal Cancer Treated with Regorafenib and Nivolumab

“…Identification of biomarkers that predict treatment benefit may assist in se-lecting patients for this strategy. Previous studies have linked liver metastasis, cytotoxic T cells, Tregs, tumorassociated macrophages, plasma levels of biomarkers of vascular biology, circulating tumor DNA, neutrophil-tolymphocyte ratio, gut microbiome, etc., with treatment response [6,9,10,[12][13][14][15]. However, predictive biomarkers for anti-angiogenesis and immunotherapies have been challenged by the limited studies and the inconsistent results.…”

“…The combination of regorafenib with PD-1 targeting for patients with refractory CRC has been evaluated in both prospective and retrospective settings [4][5][6][7][8][9][10][11][12][13][14][15]. The treatment efficacy in different studies among different patient populations varies greatly.…”