Evaluation of: Valle J, Wasan H, Palmer DH et al. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N. Engl. J. Med. 362, 1273–1281 (2010).

Biliary tract cancer is a rare disease and it is associated with a poor clinical outcome and survival. A standard therapy has not been established yet. The evaluated article reports on the first Phase III randomized controlled multicenter trial (ABC-02 trial) on palliative chemotherapy for biliary tract cancer. A total of 410 patients with locally advanced or metastatic cholangiocarcinoma, gallbladder cancer or ampullary cancer were included to receive either cisplatin followed by gemcitabine or gemcitabine alone for up to 24 weeks. The primary end point was overall survival and the secondary end point was progression-free survival. The median overall survival was 11.7 months in the cisplatin plus gemcitabine group and 8.1 months in the gemcitabine only group. The median progression-free survival was 8.0 months in the cisplatin plus gemcitabine group and 5.0 months in the gemcitabine-only group (p < 0.001). Adverse events were comparable in the two groups. Cisplatin plus gemcitabine, compared with gemcitabine alone, was associated with a significant survival advantage without an increase in substantial toxicity.