TY - JOUR
T1 - Sorafenib in patients with advanced biliary tract carcinoma
T2 - A phase II trial
AU - Bengala, C.
AU - Bertolini, F.
AU - Malavasi, N.
AU - Boni, C.
AU - Aitini, E.
AU - Dealis, C.
AU - Zironi, S.
AU - Depenni, R.
AU - Fontana, A.
AU - Del Giovane, C.
AU - Luppi, G.
AU - Conte, P.
PY - 2010/1
Y1 - 2010/1
N2 - 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.
AB - 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.
KW - Biliary tract cancer
KW - Phase II trial
KW - Sorafenib
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U2 - 10.1038/sj.bjc.6605458
DO - 10.1038/sj.bjc.6605458
M3 - Article
C2 - 19935794
AN - SCOPUS:74249098818
VL - 102
SP - 68
EP - 72
JO - British Journal of Cancer
JF - British Journal of Cancer
SN - 0007-0920
IS - 1
ER -