Get access

5-fluorouracil arterial infusion + interferon therapy for highly advanced hepatocellular carcinoma: A multicenter, randomized, phase II study


  • [Correction added after online publication 2 November 2011. Figures 2–5 were amended.]

Mr Yoshitaka Ueda, Department of Clinical Research and Development, Otsuka Pharmaceutical Co., Ltd., 3-2-27 Otedori Chuo-ku, Osaka 540-0021, Japan. Email:


Aim:  The efficacy and safety of 5-fluorouracil arterial infusion + interferon therapy (FAIT) was evaluated in patients with hepatocellular carcinoma (HCC) with a high degree of vascular invasion associated with poor prognosis, using best salvage therapy (BST) as a reference group.

Methods:  Sixty-nine patients with advanced HCC with a high degree of vascular invasion (Vp3, Vp4, Vv3) were randomly assigned to a FAIT group or a BST group. The FAIT group received interferon-α and 5-fluorouracil combination therapy; the BST group received either combination therapy of cisplatin and 5-fluorouracil (low-dose FP therapy) or cisplatin for arterial infusion.

Results:  Thirty patients in the FAIT group and 31 patients in the BST group were included in the efficacy analysis. The response rate (primary endpoint) was 26.7% (eight out of 30 patients) for the FAIT group and 25.8% (eight out of 31) for the BST group. The number of occurrences of adverse events of grade 3 or higher was 115 in 30 patients in the FAIT group and 113 in 29 patients in the BST group. None of the deaths were related to the study therapy.

Conclusions:  FAIT exerts modest antitumor effects and poses no particular safety concerns. FAIT may be a strategy of choice worth trying for advanced HCC with high degree of vascular invasion, which is associated with poor prognosis.