Department of Gastroenterology & Hepatology, AKH & Medical University of Vienna, Vienna, Austria.
Wien Klin Wochenschr. 2011 Jan;123(1-2):61-4. doi: 10.1007/s00508-010-1522-y. Epub 2011 Jan 19.
The oral multikinase inhibitor sorafenib is the new reference standard for the treatment of advanced hepatocellular carcinoma. Only few data are available on the use of sorafenib in cholangiocellular carcinoma (CCC).
A 70-year-old male patient with histologically confirmed unresectable intrahepatic cholangiocellular carcinoma not amenable to any other systemic chemotherapy was treated with sorafenib 400 mg bid.
Sorafenib treatment led to a significant improvement of tumor symptoms, liver function parameters, and a decrease in tumor marker levels. The best radiologic tumor response according to RECIST and mRECIST was stable disease (SD) with a time to progression (TTP) of 5.7 months. Side effects of sorafenib (diarrhea, fatigue, and skin toxicity) were low-grade and manageable. Twenty-four months after sorafenib initiation the patient is still alive and presents in a well-preserved physical constitution, performance status 0. Gene analyses revealed that neither B-raf nor K-ras was mutated in our patient.
Sorafenib was effective and well-tolerated in a patient with advanced cholangiocellular carcinoma. Prospective trials are warranted to evaluate the benefit of sorafenib in unresectable CCC.
口服多激酶抑制剂索拉非尼是治疗晚期肝细胞癌的新标准。关于索拉非尼在胆管细胞癌（CCC）中的应用，仅有少量数据可用。
一名 70 岁男性患者，经组织学证实为不可切除的肝内胆管细胞癌，无法接受任何其他系统化疗，采用索拉非尼 400mg bid 治疗。
索拉非尼治疗导致肿瘤症状、肝功能参数显著改善，并降低肿瘤标志物水平。根据 RECIST 和 mRECIST，最佳的影像学肿瘤反应为稳定疾病（SD），进展时间（TTP）为 5.7 个月。索拉非尼的副作用（腹泻、疲劳和皮肤毒性）为低级别且可管理。索拉非尼治疗开始后 24 个月，患者仍存活，身体状况良好，表现状态 0。基因分析显示，我们的患者既没有 B-raf 也没有 K-ras 突变。
索拉非尼在晚期胆管细胞癌患者中有效且耐受良好。需要进行前瞻性试验来评估索拉非尼在不可切除的 CCC 中的获益。
