Comparative efficacy of S-1 versus best supportive care in advanced hepatocellular carcinoma.

466 Background: S-1, an oral fluoropyrimidine derivative, has been shown to be clinically effective against hepatocellular carcinoma (HCC). We carried out a retrospective study to evaluate the efficacy of S-1, compared to best supportive care (BSC) in patients with advanced hepatocellular carcinoma. Methods: From 2009.12 to 2013.12, 32 cases of advanced hepatocellular carcinoma (the presence of extrahepatic metastasis or locally advanced disease not amenable to surgical resection or other locoregional therapies) were retrospectively analysed. 18 patients received S-1 (80 mg/m2/day, administered during days 1~14 and repeated every 21 days) and 14 patients received best supportive care (BSC). The time to progression (TTP), overall survival (OS) and safety were assessed. Results: 17 of 32 (53.1%) patients had metastatic disease, including the retroperitoneal lymph nodes (7 cases), lung (4 cases), supraclavicular lymph nodes (3 cases), abdominal wall (3 cases), brain (2 cases) and adrenal glands (2 cases). 12 patients (37.5%) had portal vein tumor thrombus. The two groups were well matched at baseline. In S-1 group,a total of 63 cycles were administered with median of 3.5 cycles (range, 2~7).The most common grade 3/4 toxicities were thrombocytopenia (28.1%), neutropenia (21.8%), elevated serum aspartate aminotransferase levels (15.6%) and rash (9.4%). A patient (5.6%) had a partial response,11 (61.1%) had stable disease, and 6 (33.3%) had progressive disease. Median TTP was 4.7 months in the S-1 group compared with 2.3 months in the BSC group (P=0.013). Overall survival was 15.1 months in patients treated with S-1, compared with 8.3 months in those who received BSC (P=0.027). Conclusions: S-1 showed an acceptable safety profile and benefit in survival in patients with advanced HCC.The conclusion needs further evaluation in randomized clinical trials.