Practicalities accessing precision medicine in biliary tract cancer

Abstract

In The Lancet Gastroenterology & Hepatology, Choong-kun Lee and colleagues1 report the results of a single-arm, phase 2 trial assessing folinic acid, fluorouracil, and oxaliplatin (FOLFOX) and trastuzumab in patients with HER2-positive advanced biliary tract cancers pre-treated with gemcitabine and cisplatin (mostly as second-line treatment). As expected, the majority of patients had a gallbladder cancer (53%) or extrahepatic cholangiocarcinoma (29%). The trial met its primary endpoint, achieving an objective response rate of 29·4% (95% CI 16·7–46·3) that surpassed the planned 25%, and excluded the null hypothesis of 10%.