Introduction: Cabozantinib has been approved by the European Medicine Agency (EMA) for hepatocellular carcinoma (HCC) previously treated with sorafenib. Cabozantinib is also being tested in combination with immune checkpoint inhibitors in the frontline setting. Real-life clinical data of cabozantinib for HCC are still lacking. Moreover, the prognostic factors for HCC treated with cabozantinib have not been investigated. Methods: We evaluated clinical data and outcome of HCC patients who received cabozantinib in the legal context of named patient use in Italy. Results: Ninety-six patients from 15 centres received cabozantinib. All patients had preserved liver function (Child-Pugh A), mostly with an advanced HCC (77.1%) in a third-line setting (75.0%). The prevalence of performance status (PS) #x3e; 0, macrovascular invasion (MVI), extrahepatic spread, and alpha-fetoprotein (AFP) #x3e;400 ng/mL was 50.0, 30.2, 67.7, and 44.8%, respectively. Median overall survival (OS) and progression-free survival were 12.1 (95% confidence interval 9.4–14.8) and 5.1 (3.3–6.9) months, respectively. Most common treatment-related adverse events (AEs) were fatigue (67.7%), diarrhoea (54.2%), anorexia (45.8%), HFSR (43.8%), weight loss (24.0%), and hypertension (24.0%). Most common treatment-related Grade 3–4 AEs were fatigue (6.3%), HFSR (6.3%), and increased aminotransferases (6.3%). MVI, ECOG-PS #x3e; 0, and AFP #x3e;400 ng/mL predicted a worse OS. Discontinuation for intolerance and no new extrahepatic lesions at the progression were associated with better outcomes. Conclusions: In a real-life Western scenario (mostly in a third-line setting), cabozantinib efficacy and safety data were comparable with those reported in its registration trial. Data regarding the prognostic factors might help in patient selection and design of clinical trials.
Liver Cancer

中文翻译：

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卡博替尼治疗不可切除肝细胞癌的真实临床数据

简介：卡博替尼已获得欧洲药品管理局 (EMA) 的批准，用于治疗既往使用索拉非尼治疗的肝细胞癌 (HCC)。卡博替尼也正在前线环境中与免疫检查点抑制剂联合进行测试。仍然缺乏卡博替尼治疗 HCC 的真实临床数据。此外，尚未研究卡博替尼治疗 HCC 的预后因素。方法：我们评估了在意大利指定患者使用的法律背景下接受卡博替尼的 HCC 患者的临床数据和结果。结果：来自 15 个中心的 96 名患者接受了卡博替尼治疗。所有患者的肝功能均保留（Child-Pugh A），大多数患者在三线治疗（75.0%）中患有晚期 HCC（77.1%）。表现状态 (PS) #x3e 的流行程度；0，大血管浸润 (MVI)、肝外扩散和甲胎蛋白 (AFP) #x3e;400 ng/mL 分别为 50.0、30.2、67.7 和 44.8%。中位总生存期（OS）和无进展生存期分别为 12.1（95% 置信区间 9.4-14.8）和 5.1（3.3-6.9）个月。最常见的治疗相关不良事件 (AE) 是疲劳 (67.7%)、腹泻 (54.2%)、厌食症 (45.8%)、HFSR (43.8%)、体重减轻 (24.0%) 和高血压 (24.0%)。最常见的与治疗相关的 3-4 级 AE 是疲劳 (6.3%)、HFSR (6.3%) 和转氨酶升高 (6.3%)。MVI，ECOG-PS #x3e；0，和法新社#x3e；400 ng/mL 预测更差的 OS。因不耐受而停药和进展时没有新的肝外病变与更好的结果相关。结论：在现实生活中的西方情景中（主要是在三线环境中），卡博替尼的疗效和安全性数据与其注册试验中报告的数据相当。有关预后因素的数据可能有助于患者选择和临床试验的设计。
肝癌