AM& A Migliavacca Center for Liver Disease, Division of Gastroenterology and Hepatology, University of Milan, Fondazione IRCCS Ca' Granda Maggiore Hospital, Milan, Italy.
Department of Internal Medicine I, University Medical Centre of the Johannes Gutenberg-University, Mainz, Germany.
Am J Transplant. 2019 Nov;19(11):3176-3184. doi: 10.1111/ajt.15551. Epub 2019 Sep 3.
Regorafenib is one option for second-line treatment of hepatocellular carcinoma (HCC), improving overall survival (OS) of sorafenib-tolerant patients who develop progression. We aim to evaluate the safety and outcomes of regorafenib as second-line treatment for HCC recurrence after liver transplantation (LT). This is a retrospective, multicenter, international study including regorafenib-treated LT patients (2015-2018), with analysis of baseline characteristics and evolutionary events during sorafenib/regorafenib treatment. Twenty-eight LT patients (57 years, 7% cirrhotics, 54% performance status 1) were included. Median time from LT to regorafenib initiation was 3.9 (1.1-18.5) years; median time on sorafenib was 11.3 (0.7-76.4) months and 14 (1-591) days from sorafenib discontinuation to regorafenib. During regorafenib (6.3 months), all patients had at least one adverse event (AE), the most common grade 3/4 AEs were fatigue (n = 7) and dermatological reaction (n = 5). While no liver rejection was observed, plasma levels of immunosuppressive drugs increased in five. Twenty-four patients developed progression (38% extrahepatic growth, 33% new extrahepatic lesions/vascular invasion). Median OS from regorafenib initiation was 12.9 (95% CI, 6.7-19.1) and 38.4 months (95% CI, 18.5-58.4) for the sorafenib initiation. This is the first study showing safety of regorafenib after LT, thus providing the rational of considering regorafenib in the clinical decision-making in sorafenib-tolerant patients with HCC recurrence after LT.
瑞戈非尼是索拉非尼耐药后进展的肝细胞癌(HCC)二线治疗的选择之一,可改善总生存期(OS)。我们旨在评估瑞戈非尼作为肝移植(LT)后 HCC 复发的二线治疗的安全性和结果。这是一项回顾性、多中心、国际性研究,纳入了接受瑞戈非尼治疗的 LT 患者(2015-2018 年),分析了索拉非尼/瑞戈非尼治疗期间的基线特征和演变事件。纳入 28 例 LT 患者(57 岁,7%肝硬化,54%体力状态 1)。从 LT 开始使用瑞戈非尼的中位时间为 3.9(1.1-18.5)年;中位索拉非尼使用时间为 11.3(0.7-76.4)个月,从停止索拉非尼到开始使用瑞戈非尼的中位时间为 14(1-591)天。在瑞戈非尼治疗期间(6.3 个月),所有患者均至少出现 1 次不良事件(AE),最常见的 3/4 级 AE 为疲劳(n=7)和皮肤反应(n=5)。虽然没有观察到肝排斥反应,但 5 例患者的免疫抑制药物血浆水平升高。24 例患者发生进展(38%肝外生长,33%新肝外病变/血管侵犯)。从瑞戈非尼开始治疗的中位总生存期为 12.9(95%CI,6.7-19.1)和 38.4 个月(95%CI,18.5-58.4)。这是第一项研究表明瑞戈非尼在 LT 后的安全性,从而为 LT 后 HCC 复发的索拉非尼耐药患者的临床决策中考虑瑞戈非尼提供了依据。