Effectiveness and safety of sorafenib for unresectable and advanced intrahepatic cholangiocarcinoma: A pilot study.

Abstract

405 Background: Patients with unresectable and advanced intrahepatic cholangiocarcinoma (ICC) usually have short survival due to a lack of effective treatment. This multicenter, single arm, open labeled, prospective study was conducted to evaluate the effectiveness and safety of the multikinase inhibitor, sorafenib, combined with best supportive care (BSC) in these patients. Methods: Forty-four patients with unresectable and advanced ICC were treated with sorafenib (400 mg, twice daily) and BSC. The primary endpoint was disease control rate (DCR) at week 12; secondary endpoints included time to progression (TTP), progression-free survival (PFS), overall survival (OS), duration of therapy (DOT), and adverse events (AEs). Results: The DCR at week 12 was 15.9%. The median TTP was 5.6 months, and the median PFS and OS were 3.2 and 5.7 months (95% confidence interval [CI]: 2.4-4.1 months; 3.7-8.5 months), respectively. The median DOT was 1.8 months (95% CI: 1.9-3.9 months). Grades 1 and 2 AEs occurred in 75% of patients, and one grade 4 AE (hand and foot skin reaction) was observed in one patient. Conclusions: This is the first and only prospective pilot study to examine the use of sorafenib specifically for ICC. Sorafenib with BSC had an acceptable DCR and safety profile in patients with unresectable and advanced ICC. [Table: see text]