Multinational phase 3 study of durvalumab plus chemotherapy for advanced biliary tract cancer

Patients with advanced biliary tract cancer (aBTC) have a poor prognosis. With standard first-line therapy (gemcitabine plus cisplatin) the median overall survival is 11.7 months, and the two-year OS rate is 15%. BTC exhibits immunogenic features including expression of PD-L1. The multinational phase 3 TOPAZ-1 trial evaluated addition of durvalumab (anti-PD-L1) versus placebo to gemcitabine and cisplatin. Both overall survival and progression-free survival were significantly superior with durvalumab versus placebo. Incidences of grade 3 or 4 adverse events were 75.7% and 77.8% with durvalumab and placebo, respectively.