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

Eur J Cancer. 2017 Dec;87:122-130. doi: 10.1016/j.ejca.2017.10.013. Epub 2017 Nov 14.


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.

Keywords: Biliary tract carcinoma; Cholangiocarcinoma; Molecular screening; Precision medicine; Targeted therapy.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Agents / therapeutic use*
  • Biliary Tract Neoplasms / drug therapy*
  • Biliary Tract Neoplasms / genetics*
  • Biliary Tract Neoplasms / mortality
  • Biliary Tract Neoplasms / pathology
  • Biomarkers, Tumor / genetics*
  • Biopsy
  • Disease-Free Survival
  • Female
  • Genetic Predisposition to Disease
  • High-Throughput Nucleotide Sequencing*
  • Humans
  • Male
  • Middle Aged
  • Molecular Diagnostic Techniques*
  • Phenotype
  • Precision Medicine*
  • Predictive Value of Tests
  • Prospective Studies
  • Risk Factors
  • Time Factors
  • Treatment Outcome


  • Antineoplastic Agents
  • Biomarkers, Tumor

Associated data