Phase 1b study of neoadjuvant cabozantinib and nivolumab for locally advanced hepatocellular carcinoma

A potentially curative hepatic resection is the optimal treatment for hepatocellular carcinoma, but most patients are no candidates voor resection. A phase 1b study at Johns Hopkins University School of Medicine evaluated the feasilibity of neoadjuvant cabozantinib combined with nivolumab for borderline resectable HCC. Of 15 patients enrolled, 12 (80%) underwent successful R0 resection, and 5 out of 12 (42%) had major or complete pathologic responses. After median one year of follow-up 4 of these 5 responders were without recurrence. The authors conclude that combination of targeted therapy and immune checkpoint inhibition is feasible in the neoadjuvant setting of HCC, and may result in pathologic responses and long-term disease-free survival in patients outside traditional resection criteria.