Multicenter phase 2 trial of first-line trastuzumab plus gemcitabine-cisplatin for HER2-positive biliary tract adenocarcinoma

HER2 overexpression has been observed in 4% to 16% of biliary tract cancers (BTCs). Trastuzumab (anti-HER2) improved outcomes in HER2-positive breast cancer and gastric cancer. A multicenter phase 2 trial in India evaluated trastuzumab plus standard gemcitabine-cisplatin therapy for previously untreated HER2-positive BTC. Disease control rate was 80%, and median progression-free and overall survival were 7 months and 10 months, respectively. The authors conclude that among BTC patients, first-line trastuzumab combined with gemcitabine-cisplatin was safe and demonstrated significant efficacy.