Biliary tract cancers: systemic therapy for advanced disease

Biliary tract carcinomas (BTC) present with minimal symptoms and thus they are often diagnosed in advanced stages that require systemic therapy. Unfortunately, if not resected, BTC's prognosis is generally poor, in part due to limited therapeutic options. Herein we will highlight the various systemic therapies that have proven efficacy in these diseases in both 1st and 2nd line. As it stands now, the combination of Gemcitabine and Cisplatin is the gold standard. Gemcitabine plus Cisplatin (Gem-Cis)-nab-paclitaxel showed improved survival in a phase II trial compared with historical controls. SWOG 1815 is a phase III trial currently underway comparing Gem-Cis-nab-paclitaxel to Gem-Cis and if positive, this has the potential to establish a new standard of care. New data from the ABC-06 study has shown a survival benefit using FOLFOX in the 2nd line setting. Molecularly targeted agents in BTC have demonstrated potential beyond Gem-Cis and while currently limited to second- and later-line therapies, ongoing trials are testing their efficacy even in newly diagnosed patients. With both incremental improvements in existing therapies and the development of entirely novel agents, the future of systemic therapy for BTC is promising.