Pembrolizumab monotherapy demonstrated durable anti-tumor activity and promising overall survival in patients with advanced hepatocellular carcinoma (HCC), according to study data presented at Gastrointestinal Cancers Symposium.
In the Phase 2 KEYNOTE-224 trial, 51 patients with advanced HCC and no prior systemic therapy were treated with pembrolizumab for approximately 2 years or until disease progression, unacceptable toxicity, consent withdrawal, or investigator decision. The overall response rate was 16%, and the disease control rate was 57%. The median time to progression was 4 months, progression-free survival (PFS) was 4 months, and median overall survival was 17 months.
At 18 months, the PFS rate was 16% and the OS rate was 46%.
Overall, 53% of patients experienced treatment-related adverse events, with diarrhea, fatigue, hypothyroidism, and myalgia the most commonly reported. Seven (14%) patients experienced Grade 3 treatment-related adverse events, and 6% of patients discontinued treatment due to treatment-related adverse events. Eleven (22%) patients had immune-mediated adverse events and infusion reactions. There was 1 treatment-related death due to myocarditis, with associated immune-related hepatitis C.
The authors concluded that pembrolizumab-based treatments should be further evaluated in HCC in the frontline setting.
Van Laethem J-L, et al. Pembrolizumab (pembro) monotherapy for previously untreated advanced hepatocellular carcinoma (HCC): Phase II KEYNOTE-224 study. Presented at: Gastrointestinal Cancers Symposium (virtual meeting). Abstract 297.