Attività antitumorale del sorafenib nei pazienti con epatocarcinoma

Sorafenib is an active multikinase and multitargeted anti-angiogenic inhibitor with anti-tumor activity, approved for HCC treatment. In this study we aimed to evaluate clinical efficacy and tolerability of sorafenib and to correlate neoplastic histologic patterns with response to therapy. Untreated patients with histologically proven, advanced HCC received oral sorafenib 400 mg/bid. Tumor response rate was assessed using Modified Response Evaluation Criteria in Solid Tumors and tolerability using the National Cancer Institute Common Terminology Criteria for Adverse Events. Immunohistochemistry was performed with specific antibodies against EGFR, RAS, RAF-1, p70s, HIF-alfa. 13 patients were included and 3 patients are still ongoing. Median age was 73 years, male/female 12/1; 15% had ECOG performance status of 0. Most patients discontinued therapy within 3 months for progressive disease but 2 patients were long-survivors (> 30 months of treatment). Sorafenib was fairly tolerated; the most common adverse events were hand-foot syndrome and diarrhea. Dose reduction occurred in 6 patients; 2 patients were withdrawn from the study due to side effects. No significant correlation between histologic features and clinical outcomes were found. Results of this trial seem to confirm the efficacy of sorafenib with a moderate profile of tolerability. The study was limited by its small sample size, further studies are needed to identify the predictors of treatment response in HCC patients.