International impact of the Advanced Biliary Tract Cancer (ABC)-02 study.

Introduction & Objective: Prior to 2010, there was no standard chemotherapy for patients with locally advanced or metastatic biliary tract cancer. The ABC-02 trial (Valle et al New Engl J Med, 2010) established the cisplatin/gemcitabine doublet as the reference treatment option in this relatively uncommon group of cancers, whose incidence in increasing.Methods: The global impact of the ABC-02 study has been assessed through analysis of the number of citations (PubMed), incorporation into international guidelines and the integration of the cisplatin/gemcitabine regimen into ongoing clinical trials (registered on http://clinicaltrials.gov).Results: The ABC-02 publication has been cited 356 times since April 2010. The cisplatin/gemcitabine combination has been adopted in the following guidelines: British Society of Gastroenterology (BSG), European Association for the Study of the Liver (EASL), International Liver Cancer Association (ILCA) and National Comprehensive Cancer Network (NCCN, USA). Thirty-one clinical trials are open or in development consequent on findings from the ABC-02 study; these include studies using cisplatin/gemcitabine as a comparator (n=3), studies with additional therapy coupled to cisplatin/gemcitabine (n=16) and second-line studies following failure with cisplatin/gemcitabine (n=6) in advanced disease. In addition, 6 studies are registered in earlier stage disease [neoadjuvant (n=3) and adjuvant (n=3)].Conclusion: Results of the ABC-02 trial have provided an important foundation on which clinical trials, present and future, are being designed. An international collaborative group (International Biliary Tract Collaborators [IBTCC]) has been established following the results of ABC-02 and the UK is leading on other advanced biliary studies (up to ABC-08 in development) through the NCRI Hepatobiliary Subgroup. Further meaningful therapeutic advancement is dependent on a better understanding of this poor-prognosis disease in addition to finding new drug options. ABC-02 afforded the first significant practice-changing step forward in these initiatives.