Patients with advanced hepatocellular carcinoma are commonly treated with Sorafenib, however outcomes remain poor. In the CheckMate 040 trial, advanced hepatocellular carcinoma patients with or without hepatitis C or B (HCV or HBV) infection and patients with or without previous sorafenib treatment were treated with nivolumab to assess treatment safety and efficacy. In the dose-escalation phase, in which patients were treated with a dose of 0.1-10 mg/kg every 2 weeks, 46 (96%) of 48 patients discontinued treatment, 42 (88%) of them due to disease progression. 12 (25%) of 48 patients had grade 3/4 treatment-related adverse events. Three (6%) patients had treatment-related serious adverse events (pemphigoid, adrenal insufficiency and liver disorder). In the dose-expansion phase, a nivolumab dose of 3 mg/kg was selected. The objective response rate of 214 patients was 20%, including three (1%) complete responses and 39 (18%) partial responses. PD-L1 expression levels were retrospectively assessed in 174 (81%) of 214 patients. In patients with PD-L1 expression ≥1%, objective responses were observed in nine (26%) of 34 patients; in patients with PD-L1 expression ≤1%, objective responses were observed in 26 (19%) of 140 patients. These results demonstrate that nivolumab treatment induces durable objective responses in patients with advanced hepatocellular carcinoma.
El-Khoueiry AB, Sangro B, Yau T, et al., Lancet. 2017. pii: S0140-6736(17)31046-2.