Post-registration experience of nivolumab in advanced hepatocellular carcinoma: an international study

Background: Nivolumab is FDA-approved in sorafenib-experienced, advanced hepatocellular carcinoma (HCC). Post-registration data of treatment in a real-world setting is lacking. Patients and methods: We performed an international, multi-center observational study to confirm safety and efficacy of nivolumab in 233 patients treated outside clinical trials from 8 centers in North America, Europe and Asia. Results: Patients received nivolumab for Barcelona Clinic Liver Cancer (BCLC) stage C (n=191, 92.0%) and Child-Pugh (CP) A (n=158, 67.8%) or B (n=75, 32.2%) HCC as first (n=85, 36.5%) or second to fourth systemic therapy line (n=148, 63.5%). Objective response rate (ORR) was 22.4% and Disease Control Rate (DCR) was 52.1%. Median overall survival (OS) was 12.2 months (95%CI 8.4-16.0) and median progression-free survival (PFS) was 10.1 months (95%CI 6.1-14.2). Treatment-related adverse events (trAE) of Grade >2 occurred in 26 patients (11.2%). Efficacy and safety were similar across CP classes and therapy line. OS was shorter in CP-B than A (7.3 months versus 16.3 months, P<0.001) and in post-first line use (10.4 versus 16.3 months, p=0.05). Achievement of objective response predicted for improved OS (25.4 months versus 13.2 months, p