Pharmacotherapeutic options for biliary tract cancer: current standard of care and new perspectives

Roberto Filippi, Pasquale Lombardi, Virginia Quarà, Elisabetta Fenocchio, Giacomo Aimar, Michela Milanesio, Francesco Leone, Massimo Aglietta

Research output: Contribution to journalReview articlepeer-review


Introduction: Biliary tract cancer (BTC), which comprises gallbladder cancer, ampullary cancer, and cholangiocarcinoma, is a rare and heterogeneous entity, with limited approved therapeutic options. However, interest in this disease has grown exponentially in recent years, as a mounting body of evidence has shed light on the complex molecular and microenvironmental background of BTC, and clinical investigations have explored a variety of new agents and combinations, with promising results.Areas covered: This review describes the standard of care in advanced BTC and summarizes the most recent evidence available on the pharmacological treatment of resected and advanced disease, focusing on chemotherapy, targeted therapy, and immunotherapy.Expert opinion: The therapeutic armamentarium of BTC has made radical progress after almost a decade of very few positive results. Phase-III evidence now supports the use of adjuvant capecitabine after resection of localized disease, while investigations into improved regimens in the advanced setting are underway, exploring alternative options to the standard gemcitabine-cisplatin doublet. The first positive phase-III trial supports the use of the mFOLFOX6 regimen as a second-line chemotherapy. Targeted therapy against specific genomic alterations can combine with chemotherapy in specific subsets of patients. Despite recent advancements, conducting clinical trials for BTC is still a real challenge.

Original languageEnglish
Pages (from-to)2121-2137
Number of pages17
JournalExpert Opinion on Pharmacotherapy
Issue number17
Publication statusPublished - Dec 2019


  • Antineoplastic Combined Chemotherapy Protocols/therapeutic use
  • Biliary Tract Neoplasms/drug therapy
  • Cisplatin/therapeutic use
  • Clinical Trials as Topic
  • Deoxycytidine/analogs & derivatives
  • Humans
  • Immunotherapy
  • Neoplasm Staging
  • Receptor, Fibroblast Growth Factor, Type 2/antagonists & inhibitors


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