The Use of Cabozantinib in Advanced Hepatocellular Carcinoma in Hong Kong—A Territory-Wide Cohort Study

SIMPLE SUMMARY: The vascular endothelial growth factor and c-MET pathways are strongly implicated in hepatocellular carcinoma (HCC). Cabozantinib inhibits both pathways and has been approved in sorafenib-exposed advanced HCC (aHCC). We aimed to evaluate the real-life pattern of use, efficacy, and safety of cabozantinib in aHCC patients in a territory-wide study. In single-agent cabozantinib patients (n = 27), we found that 3.7% had a response, 44.4% had disease control, and the median overall survival (OS) was 8.28 months. Around 74.1% of patients had adverse events (AEs). We also did an exploratory analysis of patients who received cabozantinib as an add-on to immune-checkpoint inhibitors (ICIs) after progression on ICIs. Out of 15 such patients, 6.7% had a response and the median OS was 15.1 months, with 73.3% of patients having AEs. Overall, cabozantinib had good efficacy, survival, and safety in aHCC patients in a real-life setting. ABSTRACT: (1) Background: Cabozantinib is approved in sorafenib-exposed advanced hepatocellular carcinoma (aHCC). We evaluated the real-life pattern of use, efficacy, and tolerability of cabozantinib in aHCC. (2) Methods: This territory-wide study included consecutive aHCC patients who received cabozantinib between February 2018 and September 2020 in Hong Kong. The objective response rate (ORR), disease control rate (DCR), overall survival (OS), and adverse events (AE) were assessed. (3) Results: Overall, 42 patients were included. Approximately 83.3% had Child-Pugh A cirrhosis. About 64.3% received cabozantinib as a single agent, and the remaining 35.7% received cabozantinib as an add-on to immune checkpoint inhibitors (ICIs). For single-agent patients, the median follow-up was 6.7 months. The ORR was 3.7%, DCR was 44.4%, and the median OS was 8.28 months. About 74.1% of patients experienced any AEs with 7.4% having grade ≥3 AEs. Among patients who received prior ICIs (n = 16), the ORR was 6.3%, and the median OS was 8.28 months. An exploratory analysis of patients who received cabozantinib as an add-on to ICIs showed an ORR of 6.7% and a median OS of 15.1 months, with 73.3% having any AE and 13.3% having grade ≥3 AEs. (4) Conclusions: Cabozantinib had good anti-tumor activity, survival benefits, and acceptable tolerability in real-life aHCC patients.