Multinational phase 3 trial of first-line pembrolizumab plus chemotherapy for advanced biliary tract cancer

Biliary tract cancer has a poor prognosis. The standard-of-care treatment for advanced biliary tract cancer (aBTC) is gemcitabine and cisplatin (gem-cis) chemotherapy. The multinational phase 3 KEYNOTE-966 trial evaluated addition of pembrolizumab (pembro) to first-line gem-cis for aBTC. The patients were randomized to pembro-gem-cis (pembro group) or placebo-gem-cis (placebo group). The median overall survival was 12.7 months in the pembro group versus 10.9 months in the placebo group (HR 0.83; p=0.0034). Grade 3 to 4 adverse events were seen in 79% of patients in the pembro group and 75% of the patients in the placebo group. Six percent of patients in the pembro group and 9% of patients in the placebo group died due to adverse events, including 2% and 1%, respectively, due to treatment-related adverse events. The authors conclude that pembro-gem-cis could be a new treatment option for patients with previously untreated metastatic or unresectable BTC.