Finn RS, Ryoo BY, Merle P, et al. - In view of antitumor activity and safety of pembrolizumab shown in the phase II KEYNOTE-224 trial in previously treated patients with advanced hepatocellular carcinoma (HCC), researchers performed a randomized, double-blind, phase III study, KEYNOTE-240, to assess the effectiveness as well as safety of pembrolizumab in this patient population. They performed this study at 119 medical centers in 27 countries. They randomly allocated (at a two-to-one ratio) eligible patients with advanced HCC, previously managed with sorafenib, to receive pembrolizumab plus best supportive care (BSC) or placebo plus BSC. For pembrolizumab vs placebo, the median overall survival (OS) was 13.9 months vs 10.6 months, respectively, and the median progression-free survival (PFS) was 3.0 months vs 2.8 months at the first interim analysis and 3.0 months vs 2.8 months at the final analysis, respectively. Findings revealed that OS and PFS did not reach statistical significance per specified criteria. The observed outcomes were consistent with those of KEYNOTE-224. In this patient population, a favorable risk-to-benefit ratio for pembrolizumab was supported.