Phase 2 trial of pembrolizumab in sorafenib-treated and treatment-naïve advanced hepatocellular carcinoma: long-term follow-up of KEYNOTE-224

In the multinational phase 2 KEYNOTE-224 study, pembrolizumab showed durable antitumor activity and manageable safety in adult participants with sorafenib-treated (cohort 1, n=104) or treatment-naïve (cohort 2; n=51) advanced hepatocellular carcinoma (aHCC). Patients received pembrolizumab 200 mg intravenously every 3 weeks for up to 35 cycles. Results after a median follow-up of 83.0 months for cohort 1 and 58.8 months for cohort 2 have now been published. The median overall survival was 13.2 months (95% CI 9.7-15.3) for cohort 1 and 16.8 months (8.3-23.1) for cohort 2, whereas median progression-free survival was 4.9 months (3.5-7.0) and 4.3 months (2,1-7.8), respectively. Treatment-related adverse events occurred in 73.1% (grade 3-5 26.0%) of patients in cohort 1 and 54.9% (grade 3-5 15.7%) of patients in cohort 2. The authors conclude that pembrolizumab continued to show durable response and manageable safety in patients with aHCC with or without prior systemic therapy.