Phase 2 open-label study of single-agent sorafenib in treating advanced hepatocellular carcinoma in a hepatitis B-endemic Asian population

Abstract

Background: The current study was a phase 2 open-label study to evaluate the efficacy and tolerability of single-agent sorafenib in the treatment of advanced HCC patients in a hepatitis B-endemic Asian population. METHODS: Patients with advanced hepatocellular carcinoma (HCC) received sorafenib at a dose of 400 mg twice daily in 4-week cycles. Tumor response was assessed every 3 cycles using Response Evaluation Criteria in Solid Tumors criteria. RESULTS: Fifty-one patients were enrolled in the study and were treated with sorafenib for at least 12 weeks. The median age was 56 years (range, 28-79 years). Approximately 90% had hepatitis B virus-related HCC. Thirty-six (71%) patients had underlying Child-Pugh A cirrhosis, 13 (26%) Child-Pugh B, and 2 (3%) Child-Pugh C. Four (8%) patients achieved partial responses, and 9 (18%) patients had stable disease for at least 12 weeks. The median overall survival was 5 months (range, 4-17 months). Patients without extrahepatic spread, particularly without lung metastasis (P<.01), are more likely to benefit from sorafenib treatment. The most common toxicities were diarrhea (67%), malaise (55%), and hand-foot-skin reaction (54%). The majority of patients had transient liver function derangement. Patients with and without underlying portal vein thrombosis had similar therapeutic benefits and likewise shared a similar treatment-related toxicity profile with sorafenib treatment. CONCLUSIONS: Single-agent sorafenib demonstrates good efficacy and acceptable tolerability in treating an advanced HCC patient population in a hepatitis B-endemic area. The presence of lung metastasis predicts poor response to sorafenib in advanced HCC patients. © 2009 American Cancer Society.