Therapeutic efficacy of combination therapy with intra‐arterial 5‐fluorouracil and systemic pegylated interferon α‐2b for advanced hepatocellular carcinoma with portal venous invasion

BACKGROUND: The prognosis of advanced hepatocellular carcinoma (HCC) remains poor, particularly among patients with portal vein tumor thrombosis (PVTT). This study evaluated the efficacy of combined 5‐fluorouracil and pegylated interferon (PEG‐IFN) α‐2b in patients with advanced HCC. METHODS: Subjects comprised 59 HCC patients with PVTT treated using subcutaneous administration of PEG‐IFNα‐2b (50‐100 μg on day 1 of each week for 4 weeks) and intra‐arterial infusion of 5‐fluorouracil (250 mg/d for 5 hours on days 1‐5 of each week for 4 weeks). One treatment cycle lasted 4 weeks. The current therapy was discontinued in patients with progressive disease (PD). For responses other than PD, treatment was repeated for ≥1 cycle. The primary efficacy endpoint was the objective early response rate. Secondary efficacy endpoints were progression‐free survival (PFS) and overall survival. RESULTS: Objective early response rate was 73.0%. Cumulative PFS rates were 67.4% at 6 months, 30.2% at 12 months, 25.9% at 18 months, and 20.7% at 24 months. Median PFS was 9.7 months. Cumulative survival rates were 82.4% at 6 months, 73.6% at 12 months, 52.8% at 24 months, and 44.0% at 36 months. Median survival time was 29.9 months. All adverse reactions were controllable by temporary suspension of treatment. Serious complications and treatment‐related deaths were not observed. CONCLUSIONS: Although a prospective randomized controlled trial using a larger population of patients with advanced HCC is needed to evaluate combination therapy with 5‐fluorouracil and PEG‐IFNα‐2b, this new combination therapy may be useful for patients with advanced HCC. Cancer 2011. © 2011 American Cancer Society. The present study demonstrates the efficacy of hepatic arterial infusion chemotherapy (HAIC) with pegylated interferon α‐2b and 5‐fluorouracil for advanced hepatocellular carcinoma (HCC) patients. This newly developed combination therapy is likely to be useful for patients with advanced HCC, although a large‐scale randomized controlled study in comparison with sorafenib or HAIC with other regimens is needed to properly evaluate the efficacy of this therapy.