Trastuzumab plus FOLFOX for HER2-positive biliary tract cancer refractory to gemcitabine and cisplatin

Second-line FOLFOX chemotherapy showed a survival benefit compared with active symptom control for biliary tract cancer (BTC). HER2 overexpression or amplification is seen in 15% of cases of BTC. The multicenter phase 2 KCSG-HB19-14 trial in South Korea evaluated the combination of trastuzumab and FOLFOX as second- or third-line treatment for HER2-positive BTC. The combination had promising activity and acceptable toxicity. The authors conclude that the combination warrants further investigation.