Abstract:Objective: To observe the clinical efficacy and toxic effect of sorafenib in treatment of advanced cholangiocellular carcinoma (CCC). Methods: Fifteen patients with advanced CCC were enrolled and underwent continuous oral sorafenib administration at a dose of 400 mg twice daily with 3 weeks as a treatment cycle until disease progression or intolerant drug-related toxicity. The primary endpoints included response rate (RR) and the disease control rate (DCR) at 12 weeks, and the secondary endpoints included time to progression (TTP), progression-free survival (PFS), overall survival (OS), duration of treatment (DOT) and toxicity. Results: In the 15 patients, the median DOT was 3.2 (1.5-30) months, 4 cases achieved a partial response and 7 cases obtained a stable disease, according to RECIST criteria. The median TTP, PFS and OS was 3.2, 5.5 and 5.7 months, respectively. Treatment-related adverse reactions occurred in 9 cases, in which the grade 1-2 skin rash was most common toxicity reaction (33.3%), and grade 3 hand-foot syndrome occurred in one case (6.7%). Conclusion: Sorafenib monotherapy has proven efficacy and manageable toxicity in advanced CCC.