目的：观察索拉非尼治疗进展期胆管细胞型肝癌（CCC）的临床疗效及毒性反应。方法：15例进展期CCC患者口服索拉非尼400 mg，2次/d，3周为一个周期，持续用药，直至病情进展或出现不可耐受的毒性反应。观察服药12周时的有效率（RR）和疾病控制率（DCR），次要观 察指标包括至疾病进展时间(TTP)，无进展生存期（PFS），总生存期（OS），治疗持续时间（DOT）和毒性反应。结果：15例患者中位DOT是3.2（1.5~30）个月；按照RECIST 标准评价部分缓解4例，病情稳定7例，疾病进展4例；12周时RR为26.7%，DCR为73.3%；中位TTP，PFS及OS分别为3.2、5.5和5.7个月；治疗期间出现不良反应者9例，最常见的毒副反应是1~2级皮疹（33.3%），出现3级手足综合征1例（6.7%）。结论：索拉非尼对进展期CCC患者治疗有效，其不良反应可以耐受。
Clinical observation of sorafenib for advanced cholangiocellular carcinoma
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.