The efficacy of pembrolizumab administered as second-line treatment in patients with advanced liver cancer who had previously received chemotherapy including sorafenib or oxaliplatin was reported in the KEYNOTE-394 trial.

A total of 453 patients from China, Hong Kong, Korea, Malaysia, and Taiwan were enrolled in the study, which was divided into a pembrolizumab treatment arm (300 patients) and a control arm (153 patients), with the pembrolizumab treatment arm receiving 200 mg every 3 weeks.

Median overall survival was 14.6 months in the pembrolizumab treatment arm and 13 months in the control arm, a statistically significant prolongation. One-, two-, and three-year survival rates were 57%, 34.3%, and 23.4% in the pembrolizumab arm, respectively, compared to 52.9%, 24.9%, and 11% in the control arm. The response rate was 12.7% in the pembrolizumab arm, compared to 1.3% in the control arm. On the other hand, the rate of serious adverse events of grade 3 or higher was 14.3% in the pembrolizumab treatment group, compared to 5.9% in the control group, and the rate of patients who discontinued pembrolizumab treatment due to adverse events was 4%, compared to 0.7% in the control group. 

The researchers concluded that the study showed that pembrolizumab as a second-line treatment for patients with advanced liver cancer is effective in cases where standard treatment has failed, as a prolongation of survival was observed.