The effect of transition timing of regorafenib on treatment outcomes in unresectable hepatocellular carcinoma: a real-world study

Hepatol Int. 2024 Dec 2. doi: 10.1007/s12072-024-10757-4. Online ahead of print.

Abstract

Background: Regorafenib is the recommended second-line therapy for unresectable hepatocellular carcinoma (uHCC). Our study aimed to assess the effect of transition timing to second-line therapy with regorafenib on treatment outcomes in uHCC patients.

Methods: In this retrospective study, patients were categorized into prompt transition group (PT group) or delayed transition group (DT group) based on the transition timing to second-line therapy with regorafenib following the first or subsequent occurrence of progressive disease during first-line treatment. The primary endpoint was progression-free survival (PFS), and the secondary endpoints were objective response rate (ORR), disease control rate (DCR), overall survival (OS), and safety.

Results: There were 85 and 122 patients in the PT and DT groups, respectively. The PT group demonstrated significantly better median PFS [4.5 months (95% CI 3.8-6.0) vs. 3.4 months (95% CI 2.8-4.1); HR 0.641; 95% CI 0.478-0.859; p = 0.003], ORR (24.7% vs. 9.8%, p = 0.007), DCR (69.4% vs. 54.9%, p = 0.049), and median OS [17.5 months (95% CI 13.4-not reached) vs. 10.4 months (95% CI 7.9-15.6); HR 0.613; 92% CI 0.432-0.871, p = 0.006] compared to the DT group. Moreover, the overall safety profiles were comparable between groups.

Conclusion: The prompt transition to second-line therapy with regorafenib following first-line treatment progression suggests better treatment outcomes and potentially longer survival than in patients who delay the transition to second-line therapy.

Keywords: Hepatocellular carcinoma; Regorafenib; Second-line therapy; Therapeutic strategy.