Axitinib for Gemcitabine-refractory Advanced Biliary Tract Cancer: Report of 5 Cases.

BACKGROUND/AIM Vascular endothelial growth factor receptor (VEGFR) has been identified as a treatment target for biliary tract cancer (BTC) and axitinib is a selective inhibitor of vascular endothelial growth factor receptor (VEGFR)-1/2/3. This study was conducted as a preliminary evaluation of the safety and efficacy of axitinib for patients with advanced BTC. PATIENTS AND METHODS Patients refractory to gemcitabine-based regimens were administered axitinib at the dose of 5 mg twice daily. RESULTS Five patients (3 male and 2 female) with a median age of 68 years were enrolled. Although 3 patients developed treatment-related grade 3/4 adverse events (AEs), none of these patients required discontinuation of the protocol treatment due to the AEs. Partial response (PR) was achieved in 1 patient, with a 67% reduction. The response was classified as stable disease (SD) in 3 patients and as progressive disease (PD) in 1 patient. Overall survival (OS) and progression-free survival (PFS) ranged from 2.0 to 19.9 months and 1.5 to 7.4 months, respectively. CONCLUSION This preliminary study suggested that axitinib is well-tolerated and might exert promising activity in patients with BTC.