title: Effectiveness and safety of sorafenib in the treatment of unresectable and advanced intrahepatic cholangiocarcinoma: a pilot study.
abstract: 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 sorafenib combined with best supportive care (BSC) in these patients. We enrolled 44 patients with unresectable and advanced ICC who were treated with sorafenib (400 mg, twice daily) and BSC. The primary endpoint was disease control rate (DCR) at week 12, and the secondary endpoints included time to progression (TTP), progression-free survival (PFS), overall survival (OS), duration of therapy (DOT), and adverse events (AEs). Our results showed that the DCR 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). AEs of grades 1 and 2 events occurred in 75% of patients, and AE of grade 4 (severe) was observed in 1 patient. Therefore, sorafenib in combination with BSC had an acceptable DCR and safety profile in patients with unresectable and advanced ICC.
authors: Department of Biliary Surgery, The Eastern Hepatobiliary Surgery Hospital, Second Military Medical University, Shanghai, China.
authors: Xiangji
authors: X
authors: Luo
authors: Department of Hepatobiliary Surgery, The Anhui Provincial Hospital, Hefei, China.
authors: Weidong
authors: W
authors: Jia
authors: Department of Hepatobiliary Surgery, The Tongji Hospital, Huazhong University of Science and Technology, Wuhan, China.
authors: Zhiyong
authors: Z
authors: Huang
authors: Department of Hepatobiliary Surgery, The Jiangsu Provincial People's Hospital, Nanjing, China.
authors: Xiangcheng
authors: X
authors: Li
authors: Department of Hepatobiliary Surgery, The Beijing Cancer Hospital, Beijing, China.
authors: Baocai
authors: B
authors: Xing
authors: Department of Biliary Surgery, The Eastern Hepatobiliary Surgery Hospital, Second Military Medical University, Shanghai, China.
authors: Xiaoqing
authors: X
authors: Jiang
authors: Department of Hepatic Surgery, The Eastern Hepatobiliary Surgery Hospital, Second Military Medical University, Shanghai, China.
authors: Jun
authors: J
authors: Li
authors: Department of Hepatic Surgery, The Eastern Hepatobiliary Surgery Hospital, Second Military Medical University, Shanghai, China.
authors: Anfeng
authors: A
authors: Si
authors: Department of Hepatic Surgery, The Eastern Hepatobiliary Surgery Hospital, Second Military Medical University, Shanghai, China.
authors: Tian
authors: T
authors: Yang
authors: Department of Laboratory Diagnosis, The Eastern Hepatobiliary Surgery Hospital, Second Military Medical University, Shanghai, China.
authors: Chunfang
authors: C
authors: Gao
authors: Department of Hepatic Surgery, The Eastern Hepatobiliary Surgery Hospital, Second Military Medical University, Shanghai, China.|Faculty of Medicine, The Chinese University of Hong Kong, Shatin, Hong Kong, SAR, China.
authors: Wan Yee
authors: WY
authors: Lau
authors: Department of Hepatic Surgery, The Eastern Hepatobiliary Surgery Hospital, Second Military Medical University, Shanghai, China.
authors: Feng
authors: F
authors: Shen
mesh_terms: D000368:Aged; D001650:Bile Duct Neoplasms / Q000188:drug therapy*; D001653:Bile Ducts, Intrahepatic / Q000187:drug effects* / Q000473:pathology; D018281:Cholangiocarcinoma / Q000188:drug therapy*; D003967:Diarrhea / Q000139:chemically induced; D004334:Drug Administration Schedule; D005076:Exanthema / Q000139:chemically induced; D005221:Fatigue / Q000139:chemically induced; D005260:Female; D006801:Humans; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D009536:Niacinamide / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010166:Palliative Care; D010671:Phenylurea Compounds / Q000009:adverse effects / Q000627:therapeutic use*; D010865:Pilot Projects; D011446:Prospective Studies; D047428:Protein Kinase Inhibitors / Q000009:adverse effects / Q000627:therapeutic use; D000077157:Sorafenib; D016896:Treatment Outcome
chemical_list: D010671:Phenylurea Compounds; D047428:Protein Kinase Inhibitors; D009536:Niacinamide; D000077157:Sorafenib
keywords: adverse events; disease control rate; intrahepatic cholangiocarcinoma; sorafenib