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

Abstract

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 (CI_95% 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.

Keywords:

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

Acknowledgements

The authors acknowledge all the Cholangiocarcinoma Italian Group Onlus (G.I.C.O.) investigators for their contribution and support to the present work.

Disclosure statement

No potential conflict of interest was reported by the authors.

Data availability statement

The dataset is available upon request addressed to the corresponding author.

Author contributions

(I) Conception and design: Roberto Filippi

(II) Administrative support: Roberto Filippi

(III) Provision of study materials or patients: All authors

(IV) Collection and assembly of data: All authors

(V) Data analysis and interpretation: Roberto Filippi and Francesco Leone

(VI) Manuscript writing: Roberto Filippi, Francesco Leone, Pasquale Lombardi, Giovanni Brandi

(VII) Final approval of manuscript: All authors

The authors have completed the STROBE reporting checklist.

Ethics approval

This study received ethics approval (protocol n° 0072135).