Clinical insights and prognostic factors from an advanced biliary tract cancer case series: a real-world analysis.

Roberto Filippi, Francesco Leone, Lorenzo Fornaro, Giuseppe Aprile, Andrea Casadei-Gardini, Nicola Silvestris, Andrea Palloni, Maria Antonietta Satolli, Mario Scartozzi, Marco Russano, Stefania Eufemia Lutrino, Pasquale Lombardi, Giorgio Frega, Silvio Ken Garattini, Caterina Vivaldi, Rosella Spadi, Orsi Giulia, Elisabetta Fenocchio, Oronzo Brunetti, Massimo AgliettaGiovanni Brandi

Research output: Contribution to journalArticlepeer-review


Advanced biliary tract cancer (aBTC) comprises a heterogeneous group of rare malignancies with dismal prognosis. Given the scarcity of prospective evidence, the aim of this study was to derive clinically useful insights and prognostic factors from a large, real-world series of aBTC. Clinicopathologic variables and treatment outcomes were retrospectively collected involving 940 patients diagnosed with aBTC between 2001 and 2017, and treated with first-line chemotherapy (CT1) at 14 Italian medical oncology institutions. Median overall survival (OS) was 10.3 months (CI95% 9.5-11.1). CT1 with gemcitabine-Platinum salts doublets achieved OS of 11.7 months vs 7.5 with gemcitabine alone (HR 0.67, p < 0.001). However, a clear temporal trend towards improved OS could not be demonstrated. Radical surgery of recurrent disease achieved a relapse-free survival of 5.9 months. A substantial minority (44.5%) of patients were able to receive a second-line chemotherapy, which achieved a response rate of 7.6%, and disease control in 30% of patients with no significant differences between combination regimens and monotherapies. In a large retrospective series of real-world aBTC, outcomes of standard CT1 closely resembled those of the registrational trials. A limited set of easily retrievable independent prognostic factors was defined. Further research is needed on second-line regimens.

Original languageEnglish
JournalJournal of Chemotherapy
Publication statusAccepted/In press - 2021


  • Advanced biliary tract neoplasms
  • ampullary neoplasms
  • chemotherapy
  • extrahepatic cholangiocarcinoma
  • gallbladder neoplasms
  • intrahepatic cholangiocarcinoma
  • prognosis

ASJC Scopus subject areas

  • Oncology
  • Pharmacology
  • Pharmacology (medical)
  • Infectious Diseases


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