Pembrolizumab

In an international, phase II, open-label, single-arm study (KEYNOTE-224 [NCT02702414]), 104 patients with BCLC stage B or C disease were enrolled across Europe, North America, and Japan. Patients had advanced HCC refractory to, or intolerant of, sorafenib and received pembrolizumab (200 mg IV every 3 weeks).[23]

In an international, phase II, open-label, single-arm study (KEYNOTE-224 [NCT02702414]), 104 patients with BCLC stage B or C disease were enrolled across Europe, North America, and Japan. Patients had advanced HCC refractory to, or intolerant of, sorafenib and received pembrolizumab (200 mg IV every 3 weeks).[23]

The objective response rate was 18.3% (95% CI, 11.4%–27.1%), and the median duration of response was 21.0 months (range, 3.1–39.5+).[23,24]

In an international, phase II, open-label, single-arm study (KEYNOTE-224 [NCT02702414]), 104 patients with BCLC stage B or C disease were enrolled across Europe, North America, and Japan. Patients had advanced HCC refractory to, or intolerant of, sorafenib and received pembrolizumab (200 mg IV every 3 weeks).[23]

The median OS was 13.2 months (95% CI, 9.7–15.3).

The phase III KEYNOTE-394 study (NCT03062358) included 453 patients in Asia with advanced HCC previously treated with sorafenib or oxaliplatin-based chemotherapy. Patients were randomly assigned in a 2:1 ratio to receive either pembrolizumab (200 mg IV) or placebo every 3 weeks for up to 35 cycles.[25]

The phase III KEYNOTE-394 study (NCT03062358) included 453 patients in Asia with advanced HCC previously treated with sorafenib or oxaliplatin-based chemotherapy. Patients were randomly assigned in a 2:1 ratio to receive either pembrolizumab (200 mg IV) or placebo every 3 weeks for up to 35 cycles.[25]

The OS was 14.6 months (95% CI, 12.6–18.0) in the pembrolizumab arm and 13.0 months (95% CI, 10.5–15.1) in the placebo arm (HR, 0.79; 95% CI, 0.63–0.99; P = .0180). Notably, the 24-month OS rate was 34.3% in the pembrolizumab arm and 24.9% in the placebo arm.[25][Level of evidence A1]

The phase III KEYNOTE-394 study (NCT03062358) included 453 patients in Asia with advanced HCC previously treated with sorafenib or oxaliplatin-based chemotherapy. Patients were randomly assigned in a 2:1 ratio to receive either pembrolizumab (200 mg IV) or placebo every 3 weeks for up to 35 cycles.[25]

Grade 3 or higher treatment-related adverse events occurred in 14.4% of patients in the pembrolizumab arm and 5.9% of patients in the placebo arm.