Safety 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 with best supportive care (BSC) in LT patients after sorafenib discontinuation. This observational multicenter retrospective study included LT patients with HCC recurrence who discontinued first-line sorafenib. Group 1 comprised regorafenib-treated patients, whereas the control group was selected among patients treated with 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 with group 2. Secondary endpoints were safety and OS of sequential treatment with sorafenib + regorafenib/BSC. Among 132 LT patients who discontinued sorafenib included in the study, 81 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 versus 5.5 months; P < 0.01). Regorafenib treatment was an independent predictor of reduced mortality (hazard ratio, 0.37; 95% confidence interval [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% (n = 28). The median OS calculated from sorafenib start was 28.8 months (95% CI, 17.6-40.1) in group 1 versus 15.3 months (95% CI, 8.8-21.7) in group 2 (P < 0.01). Regorafenib is an effective second-line treatment after sorafenib in patients with HCC recurrence after LT.

中文翻译：

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肝移植后复发性肝细胞癌患者接受索拉非尼治疗后瑞戈非尼的疗效：一项回顾性研究

最近证明了瑞格非尼在肝移植 (LT) 后肝细胞癌 (HCC) 复发中的安全性。我们旨在评估在索拉非尼停药后 LT 患者中瑞戈非尼与最佳支持治疗 (BSC) 相比的生存获益。这项观察性多中心回顾性研究包括停止一线索拉非尼治疗的 HCC 复发 LT 患者。第 1 组包括接受瑞戈非尼治疗的患者，而对照组是在接受 BSC 治疗的患者中选择的，因为在索拉非尼停药时无法获得二线选择，并且是索拉非尼耐受的进展者（第 2 组）。主要终点是第 1 组与第 2 组相比的总生存期 (OS)。次要终点是索拉非尼 + 瑞戈非尼 / BSC 序贯治疗的安全性和 OS。在研究中包括停止索拉非尼治疗的 132 名 LT 患者中，81 名患者耐受索拉非尼：36 名接受瑞戈非尼（第 1 组），45 名（第 2 组）接受 BSC。总体而言，第 1 组中有 24 名 (67%) 患者死亡，第 2 组中有 40 名 (89%) 患者死亡：第 1 组的中位 OS 显着长于第 2 组（13.1 个月 vs 5.5 个月；P  < 0.01)。瑞戈非尼治疗是降低死亡率的独立预测因子（风险比，0.37；95% 置信区间 [CI]，0.16-0.89；P  = 0.02）。瑞戈非尼的中位治疗持续时间为 7.0 (95% CI, 5.5-8.5) 个月；22 名 (61%) 患者因不良事件减少瑞戈非尼剂量，93% (n = 28) 因肿瘤进展停止服用。从索拉非尼开始计算的中位 OS，第 1 组为 28.8 个月（95% CI，17.6-40.1），第 2 组为 15.3 个月（95% CI，8.8-21.7）（P  < 0.01）。对于 LT 后 HCC 复发的患者，瑞戈非尼是索拉非尼后有效的二线治疗药物。