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

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.

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.

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.

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.