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Real-world outcomes in metastatic colorectal patients receiving regorafenib treatment in China
Background
Regorafenib has been recognized as a standard third-line treatment in mCRC, while real-world data of large samples and treatment modalities in China are limited. In addition, the optimal dosage of regorafenib, and the efficacy and related factors of regorafenib combination therapy, still need to be elucidated.
Methods
We conducted a retrospective real word study for Chinese patients (Pts) with refractory metastatic colorectal (mCRC) received at least one cycle of regorafenib as the third or higher line treatment (40, 80, 120, or 160mg QD for 21 days, as a 28-day cycle) from August 2017 to June 2020. Data of demographic, treatment modality, dosimetry, and survival were collected. In addition, univariate and multivariate analyses were conducted based on Kaplan-Meier analysis and Cox regression model.
Results
713 Pts ever received regorafenib from 10 centers were screened, and 598 Pts were finally enrolled with a median age of 59 years old (18-84). In these Pts, 364 (60.87%) received regorafenib monotherapy, and 234 (39.13%) accepted combination therapy, most (82.27%) as the third or fourth line treatment. The maintenance dose was confirmed in 380 Pts, among whom 164 (43.15%) and 118 (31.05%) accepted 80 and 120mg as the final maintenance dose. With a median follow-up time of 8.77 months, the median PFS was 4.67 months (95%CI: 3.98-5.55), and median OS was 16.33 months (95% CI: 13.08-20.17). In univariate analysis, the dosage of regorafenib in maintenance treatment was significantly associated with median OS. The maintenance dose's median OS of 40, 80, 120, or 160mg were 4.57, 12.19, 16.23, and 25.10 months, respectively (p=0.0027). In multivariate analysis, Pts accepted 40, and 80mg still had inferior survivals, whereas no difference was observed over 120mg (HR=1.542, 95%CI 0.880-2.701; p=0.1302). Moreover, compared to regorafenib monotherapy, combination therapy of regorafenib and PD-1 inhibitor with or without chemotherapy achieved a longer survival in univariate and multivariate analysis (p=0.0485, p=0.0189, respectively). While regorafenib plus PD-1 inhibitor duplet regimen only achieved a numerically longer median OS in the univariate analysis compared to regorafenib monotherapy (18.27m vs. 13.08m, p=0.2353).
Conclusions
Regorafenib showed satisfactory efficacy as a later-line treatment in Chinese mCRC patients in real-world practice, with a recommended maintenance dose no less than 120mg. The modality of combination therapy containing regorafenib and PD-1 inhibitor yielded encouraging overall survival, and further investigation is warranted.
Clinical trial identification
Clinical trial information: NCT04735991.
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosures
All authors have declared no conflicts of interest.
