A multi-institutional phase II trial of hepatic arterial infusion chemotherapy with cisplatin for advanced hepatocellular carcinoma with portal vein tumor thrombosis.

PURPOSE: The objective of this study was to evaluate the response rate, survival, and adverse effects of hepatic arterial infusion chemotherapy (HAIC) using cisplatin in patients with advanced hepatocellular carcinoma (HCC) and portal vein tumor thrombosis (PVTT).

METHODS: Twenty-five patients of advanced HCC with PVTT in the main or first branch, having no prior history of chemotherapy, measurable lesions, adequate liver and renal function, and adequate bone marrow reserve, were enrolled. Cisplatin was administered at the dose of 65 mg/m(2) via the proper hepatic artery. Treatment was repeated every 4-6 weeks for a maximum of six courses until the appearance of evidence of tumor progression or unacceptable toxicity.

RESULTS: The median number of treatments was 3 (range 1-6). Among the 25 enrolled patients, complete response was achieved in 1 (4 %) patient and partial response in 6 (24 %), corresponding to a response rate of 28 % (95 % CI 12-49 %). The median progression-free and overall survival times and the 1-, 2-, and 3-year survival rates in the enrolled patients were 3.6 and 7.6 months and 40.3, 36.0, 20 %, respectively. Four of the seven patients who showed complete or partial response survived for more than 3 years. The main grade 3/4 non-hematological adverse events of this treatment were elevation of the serum aspartate aminotransferase (44 %) and alanine aminotransferase (24 %).

CONCLUSION: HAIC with cisplatin exerts moderate activity with mild toxicity in advanced HCC patients with PVTT. Especially, markedly prolonged survival can be expected in patients who respond to this treatment.