Sorafenib in patients with advanced biliary tract carcinoma: A phase II trial

C. Bengala, F. Bertolini, N. Malavasi, C. Boni, E. Aitini, C. Dealis, S. Zironi, R. Depenni, A. Fontana, C. Del Giovane, G. Luppi, P. Conte

Research output: Contribution to journalArticlepeer-review


Background: Advanced biliary tract carcinoma has a very poor prognosis, with chemotherapy being the mainstay of treatment. Sorafenib, a multikinase inhibitor of VEGFR-2/-3, PDGFR-Β, B-Raf, and C-Raf, has shown to be active in preclinical models of cholangiocarcinoma.Methods: We conducted a phase II trial of single-agent sorafenib in patients with advanced biliary tract carcinoma. Sorafenib was administered at a dose of 400 mg twice a day. The primary end point was the disease control rate at 12 weeks.Results: A total of 46 patients were treated. In all, 26 (56%) had received chemotherapy earlier, and 36 patients completed at least 45 days of treatment. In intention-to-treat analysis, the objective response was 2% and the disease control rate at 12 weeks was 32.6%. Progression-free survival (PFS) was 2.3 months (range: 0-12 months), and the median overall survival was 4.4 months (range: 0-22 months). Performance status was significantly related to PFS: median PFS values for ECOG 0 and 1 were 5.7 and 2.1 months, respectively (P0.0002). The most common toxicities were skin rash (35%) and fatigue (33%), requiring a dose reduction in 22% of patients.Conclusions: Sorafenib as a single agent has a low activity in cholangiocarcinoma. Patients having a good performance status have a better PFS. The toxicity profile is manageable.

Original languageEnglish
Pages (from-to)68-72
Number of pages5
JournalBritish Journal of Cancer
Issue number1
Publication statusPublished - Jan 2010


  • Biliary tract cancer
  • Phase II trial
  • Sorafenib

ASJC Scopus subject areas

  • Cancer Research
  • Oncology


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