Real-Life Clinical Data of Cabozantinib for Unresectable Hepatocellular Carcinoma

Francesco Tovoli, Vincenzo Dadduzio, Stefania De Lorenzo, Lorenza Rimassa, Gianluca Masi, Massimo Iavarone, Fabio Marra, Ingrid Garajova, Maria Pia Brizzi, Bruno Daniele, Franco Trevisani, Carlo Messina, Francesco Di Clemente, Sara Pini, Giuseppe Cabibbo, Alessandro Granito, Mario Domenico Rizzato, Vittorina Zagonel, Giovanni Brandi, Tiziana PressianiPiera Federico, Caterina Vivaldi, Irene Bergna, Claudia Campani, Fabio Piscaglia

Research output: Contribution to journalArticlepeer-review


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) > 0, macrovascular invasion (MVI), extrahepatic spread, and alpha-fetoprotein (AFP) >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 > 0, and AFP >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.

Original languageEnglish
Pages (from-to)370–379
JournalLiver Cancer
Publication statusPublished - 2021


  • Cabozantinib
  • Hepatocellular carcinoma
  • Outcome
  • Sorafenib
  • Tyrosine kinase inhibitors

ASJC Scopus subject areas

  • Hepatology
  • Oncology


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