Background: 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).
Methods: 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.
Results: 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.
Conclusions: 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.