Background and Aims: The multikinase inhibitor cabozantinib has been approved for hepatocellular carcinoma (HCC) previously treated with sorafenib. We report safety and efficacy data of an international, multicenter, real-life cohort of patients with advanced HCC treated with cabozantinib. Methods: Patients with HCC who were treated with cabozantinib were retrospectively identified across 11 centers in Austria, Switzerland, and Germany. Patients’ characteristics, adverse events, duration of treatment and overall survival (OS) data were analyzed until April 1, 2020. Results: Eighty-eight patients from 11 centers were included. The predominant underlying liver diseases were NAFLD/NASH in 26 (30%) and hepatitis C infection in 21 (24%) patients. Seventy-eight patients (89%) were classified as Barcelona clinic liver cancer (BCLC) stage C. Sixty patients (68%) were Child-Pugh A, whereas 22 (25%) were Child-Pugh B, respectively. Cabozantinib was used as systemic second- and third-line or later treatment in 41 (47%) and 46 (52%) patients, respectively. The following best responses under cabozantinib were documented: partial response in 6 (7%), stable disease in 28 (32%), and progressive disease in 28 (32%) patients, respectively. Fifty-two patients (59%) died during follow-up. The median OS from start of cabozantinib treatment was 7.0 months in the entire cohort and 9.7 months in Child-Pugh A patients, while Child-Pugh B patients had a median OS of 3.4 months, respectively. Thirty-seven (42%) patients fulfilled the CELESTIAL inclusion and exclusion criteria, showing a median OS of 11.1 months. Most common adverse events were fatigue (15.6%) and diarrhea (15.6%). Conclusion: Cabozantinib treatment was effective, safe, and feasible in patients with advanced HCC in patients with compensated cirrhosis. Patients in the real-life setting had more advanced liver disease – in which 25% of patients were Child-Pugh B. However, OS in patients with Child-Pugh A cirrhosis was similar to that reported in the phase 3 trial (CELESTIAL).
Liver Cancer

中文翻译：

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卡博替尼治疗晚期肝细胞癌：来自国际多中心真实队列的疗效和安全性数据


背景和目的：多激酶抑制剂卡博替尼已被批准用于先前接受索拉非尼治疗的肝细胞癌（HCC）。我们报告了国际、多中心、现实生活中接受卡博替尼治疗的晚期 HCC 患者队列的安全性和有效性数据。方法：对奥地利、瑞士和德国 11 个中心接受卡博替尼治疗的 HCC 患者进行回顾性鉴定。分析截至 2020 年 4 月 1 日的患者特征、不良事件、治疗持续时间和总生存 (OS) 数据。结果：纳入来自 11 个中心的 88 名患者。 26 名患者 (30%) 的主要潜在肝脏疾病为 NAFLD/NASH，21 名患者 (24%) 的主要潜在肝脏疾病为丙型肝炎感染。 78 名患者 (89%) 被分类为巴塞罗那诊所肝癌 (BCLC) C 期。60 名患者 (68%) 为 Child-Pugh A 期，22 名患者 (25%) 为 Child-Pugh B 期。卡博替尼分别被 41 名 (47%) 和 46 名 (52%) 患者用作全身二线和三线或更晚治疗。记录了卡博替尼治疗下的以下最佳反应：分别有 6 名患者 (7%) 部分缓解、28 名患者 (32%) 疾病稳定、28 名患者 (32%) 疾病进展。 52 名患者 (59%) 在随访期间死亡。整个队列中从卡博替尼治疗开始的中位 OS 为 7.0 个月，Child-Pugh A 患者为 9.7 个月，而 Child-Pugh B 患者的中位 OS 为 3.4 个月。 37 名 (42%) 患者符合 CELESTIAL 纳入和排除标准，中位 OS 为 11.1 个月。最常见的不良事件是疲劳（15.6%）和腹泻（15.6%）。 结论：卡博替尼治疗代偿期肝硬化晚期 HCC 患者有效、安全、可行。现实生活中的患者患有更晚期的肝病，其中 25% 的患者属于 Child-Pugh B 型。然而，Child-Pugh A 型肝硬化患者的 OS 与 3 期试验 (CELESTIAL) 中报告的相似。
 肝癌