Precision medicine for patients with advanced biliary tract cancers: An effective strategy within the prospective MOSCATO-01 trial

Loic Verlingue, David Malka, Adrien Allorant, Christophe Massard, Charles Ferté, Ludovic Lacroix, Etienne Rouleau, Nathalie Auger, Maud Ngo, Claudio Nicotra, Thierry De Baere, Lambros Tselikas, Bakar Ba, Stefan Michiels, Jean Yves Scoazec, Valérie Boige, Michel Ducreux, Jean Charles Soria, Antoine Hollebecque

Research output: Contribution to journalArticlepeer-review

104 Scopus citations


Background Recommended treatments of patients with advanced biliary tract cancer (aBTC) are limited to one chemotherapy doublet. Nevertheless, efficacy of treatment personalisation for aBTCs is supported by accumulating evidences but remains to be evaluated. Patients and methods Patients with aBTCs included in the prospective clinical trial MOSCATO-01 were treated by at least one previous systemic treatment, had an ECOG performance status of 0–1, and at least one tumour site accessible to biopsy. Multiple high-throughput molecular analysis was performed on biopsies to drive the administration of molecular targeted agents (MTAs). Results From November 2011 to March 2016, 43 patients (4%) of the 1035 adult patients included in MOSCATO-01 had aBTCs with a majority of intrahepatic localisation (67%). Successful biopsy procedures and DNA extractions led to molecular portraits for 34 patients (79%). Orientation to an appropriate early clinical trial or accessible MTA(s) was possible for 23 of these patients (68%), and 18 (53%) have received matched MTA(s). Among them, the overall response rate was 33% and the disease control rate was 88%. A PFS ≥6 months was observed in 37% and the PFS ratio was >1.3 for 50% of the patients. These patients had a lower risk for death as compared to the 20 patients not orientated to a matched MTA (HR, 0.29; 95% CI, 0.11–0.76; p = 0.008). Conclusions Within the MOSCATO-01 trial, patients with aBTCs had the highest rate of orientation to matched MTAs and derived a clear clinical benefit. A broader evaluation of these findings may improve future treatments strategies for aBTCs. Trial Registration: NCT01566019.

Original languageEnglish
Pages (from-to)122-130
Number of pages9
JournalEuropean Journal of Cancer
StatePublished - Dec 2017
Externally publishedYes

Bibliographical note

Publisher Copyright:
© 2017 Elsevier Ltd


  • Biliary tract carcinoma
  • Cholangiocarcinoma
  • Molecular screening
  • Precision medicine
  • Targeted therapy


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