The CORRECT trial revealed the safety and efficacy of regorafenib for patients (pts) with colorectal cancer including Japanese. Regorafenib was approved in Japan in March 2013. However, there are few studies exploring the efficacy and safety of regorafenib in a large number of pts, particularly in daily practice. Therefore, we performed this retrospective study (HGCSG1401) in order to investigate the safety and efficacy of regorafenib for Japanese pts in dairy practice.
We analyzed 74 pts who received regorafenib from May 2013 in the multi-institutional retrospective study (HGCSG1401). This study was analyzed by CTCAE v4.0 for adverse events, RECIST v1.1 for response rate (RR), and Kaplan-Meier method for progression free survival (PFS) and overall survival (OS).
Patients' characteristics were as follows; male/female 43/31, median age 66 (range 29-87), ECOG PS (0/1/2/3) 11/51/11/1, KRAS Exon2 wild/mutant 41/32 (1 patient; KRAS Exon2 status was not tested). The initial starting dose was 160 mg (n= 57, 77.0%), 120mg (n = 16, 21.6%), and 80mg (n = 1, 1.4%) respectively. Dose reductions were required in 25 pts (33.8%); 21 pts (28.8%) discontinued therapy due to adverse events. The common grade 3 adverse events (≥15%) were palmar-plantar erythrodysesthesia syndrome (n = 20; 27.0%), hypertension (n = 18; 24.3%), and AST increased (n = 12; 16.2%). RR and disease control rate (DCR) were 1.4% and 29.0%, respectively. Median PFS and median survival time (MST) were 2.0 and 5.4 months. In an analysis on relationship between ECOG PS (PS 0-1 vs. PS 2-3) and efficacy, median PFS was 2.1 vs. 1.6 months (HR 2.200, p = 0.021), and MST was 6.3 vs. 2.3 months (HR 3.816, p < 0.001).
The efficacy of regorafenib in pts with PS 0–1 was similar to the previous report; however, regorafenib was not effective in patients with PS 2–3. In adverse events, hypertension and liver dysfunction were expressed more frequently than previously reported. This analysis had been presented at the ESMO 17th World Congress on Gastrointestinal Cancer (Yuki S, et al. Abstract number was P-271).
Clinical trial identification
All authors have declared no conflicts of interest.