Objective: Sorafenib is the only effective drug for advanced hepatocellular carcinoma (HCC), but few data predictive of its effectiveness are available. To address this issue we analyzed the relationship between risk factors associated with sorafenib treatment and overall survival (OS).
Methods: Forty patients with advanced HCC were treated with sorafenib. OS was the primary endpoint and tumor response was evaluated using the Response Evaluation Criteria in Solid Tumors. Demographics, and the efficacy and adverse effects of sorafenib were analyzed. Univariate and multivariate analysis were carried out to identify risk factors for OS. The side-effects of sorafenib were summarized, and published data related to sorafenib in patients with HCC were reviewed.
Results: The median OS was 12.7 months. A partial response was achieved in five patients and stable disease was achieved in 24 patients, with a disease-stabilization rate of 60%. Hand-foot syndrome, hypertension, diarrhea, and fatigue were common adverse effects. Univariate analysis showed that tumor/lymph node/metastasis staging, Barcelona Clinic Liver Cancer staging, distant metastasis, ascites, and portal thrombosis were risk factors for OS. Multivariate analysis demonstrated that the presence of distant metastasis and ascites predicted poorer OS, and the presence of adverse effects predicted better OS. Presence of adverse effects can be used for monitoring the efficacy of sorafenib that has not been reported in previous studies.
Conclusion: Sorafenib demonstrated good efficacy and acceptable tolerability in treating an advanced HCC patient population, with or without prior treatment. The presence of ascites or distant metastasis predicted poorer OS, and the presence of adverse effects predicted improved OS.