Regorafenib efficacy after sorafenib in patients with recurrent HCC after liver transplantation: a retrospective study.

Abstract

BACKGROUND AND AIM\nSafety of regorafenib in hepatocellular carcinoma (HCC) recurrence after liver transplantation (LT) has been recently demonstrated. We aimed to assess the survival benefit of regorafenib compared to best supportive care (BSC) in LT-patients after sorafenib discontinuation.\n\n\nMETHODS\nThis observational multicenter retrospective study included LT-patients with HCC-recurrence who discontinued first-line sorafenib. Group-1 was constituted by regorafenib-treated patients, while control group was selected among patients treated with best supportive care (BSC) due to unavailability of second-line options at the time of sorafenib discontinuation and who were sorafenib-tolerant progressors (group-2). Primary endpoint was overall survival (OS) of group-1 compared to group-2. Secondary endpoints were safety and OS of sequential treatment sorafenib+regorafenib/BSC.\n\n\nRESULTS\nAmong 132 LT-patients who discontinued sorafenib included in the study, 81 patients were sorafenib-tolerant: 36 received regorafenib (group-1) and 45 (group-2) received BSC. Overall, 24 (67%) patients died in group-1 and 40 (89%) in group-2: the median OS was significantly longer in group-1 than in group-2 (13.1 vs 5.5 months; p=0.002). Regorafenib treatment was an independent predictor of reduced mortality (HR 0.37, 95%CI 0.16-0.89, p=0.02). Median treatment duration with regorafenib was 7.0 (95%CI 5.5-8.5) months; regorafenib dose was reduced in 22 (61%) patients for adverse events and discontinued for tumor progression in 93%. The median OS calculated from sorafenib start was 28.8 months (95%CI: 17.6-40.1) in group-1 vs 15.3 months (95%CI: 8.8-21.7) in group-2 (p=0.002).\n\n\nCONCLUSIONS\nRegorafenib is an effective second-line treatment after sorafenib in patients with HCC-recurrence after LT.