Analysis of efficacy and safety for the combination of regorafenib and PD-1 inhibitor in advanced hepatocellular carcinoma:A real-world clinical study

Background and aims:Hepatocellular carcinoma(HCC)is a prevalent and deadly disease with limited treatment options.Regorafenib,a tyrosine kinase inhibitor,has shown promise in HCC treatment but faces limitations as a monotherapy.Combining regorafenib with PD-1 inhibitor may improve efficacy and survival outcomes for patients.This retrospective analysis was conducted to explore its efficacy and safety,providing reference experience for better application of this combination therapy.Methods:This retrospective single-center study evaluated the efficacy and safety of combining regorafenib with PD-1 blockade for patients with HCC.Efficacy was evaluated according to the RECIST 1.1 evaluation criteria.Safety was assessed using CTCAE 4.0.Data was analyzed to compare survival status in different subgroups.Results:Generally,there were 76 patients with HCC elected to receive the regorafenib plus PD-1 blockade treatment during the study period.The objective response rate was 21.1%(n?16),and the disease control rate was 56.6%(n?43).Median progression-free survival(PFS)was 6.8 months,and median overall survival had not yet been reached.All patients suffered of at least 1 adverse event.Grade3 adverse events occurred in 31.6%of patients(n?24),with the most common being hand-foot syndrome,decreased appetite,and abdominal distension.Subgroup analyses showed no significant differences in PFS based on cirrhosis status or previous treatment lines.Conclusion:With manageable safety,regorafenib combined PD-1 inhibitor could bring survival benefits for advanced HCC who have received systemic treatment.Further,the Cox analysis showed that HBV infection,metastasis,etc.did not have significant effects on the survival benefits.