Background: The aim of this study was to compare the efficacy of hepatic arterial infusion chemotherapy (HAIC) and sorafenib in advanced hepatocellular carcinoma (HCC) patients with portal vein tumor thrombosis (PVTT). Methods: A total of 110 patients were observed between February 2008 and May 2013 in seven Korean centers. Fifty patients were treated with HAIC, and 60 patients were treated with sorafenib. Results: The disease control rate in the HAIC was significantly higher than that in the sorafenib group (p < 0.001), although there was no significant difference in the objective response rate (p = 0.214). The median overall survival (OS) was significantly longer in the HAIC group than in the sorafenib group (7.1 vs. 5.5 months, p = 0.011). The median time to-progression (TTP) was also significantly longer in the HAIC group than in the sorafenib group (3.3 vs. 2.1 months, p = 0.034). In the multivariate analysis, tumor diameter (≥ 10 cm) and the absence of combined loco-regional treatment were significant prognostic factors influencing OS (p = 0.002 and p = 0.010, respectively) and TTP (p = 0.017 and p = 0.006, respectively). The treatment modality tended to be a significant prognostic factor for survival (p = 0.052), but not for tumor progression (p = 0.121). Conclusions: HAIC is comparable with sorafenib in terms of OS and TTP in advanced HCC patients with PVTT. HAIC shows more favorable treatment responses compared with sorafenib. Therefore, HAIC might be an alternative treatment modality to sorafenib in advanced HCC patients with PVTT.
All Science Journal Classification (ASJC) codes