A phase II trial of transcatheter arterial infusion chemotherapy with an epirubicin-Lipiodol emulsion for advanced hepatocellular carcinoma refractory to transcatheter arterial embolization

Introduction Transcatheter arterial embolization (TAE) has been recognized as an effective palliative treatment option for advanced hepatocellular carcinoma (HCC). However, no effective alternative treatments for TAE-refractory HCC have yet been established. The aim of this study was to evaluate the antitumor activity and toxicity of transcatheter arterial infusion chemotherapy using an epirubicin-Lipiodol emulsion in patients with TAE-refractory HCC. Methods Patients with TAE-refractory HCC were enrolled. A dose of 60 mg/m² epirubicin emulsified in Lipiodol and contrast medium was administered from the feeding artery of the HCC. Treatment was repeated every 4 to 12 weeks if there was no evidence of tumor progression or unacceptable toxicity. Results Twenty patients were enrolled in this trial. The median number of treatment courses was 1 (range 1–4). Among the enrolled patients, one (5%) achieved a partial response, and three (15%) showed a minor response. Five (25%) patients had no change and 11 (55%) showed progressive disease. The median survival time, 1-year survival rate and median progression-free survival time for the patients as a whole were 12.4 months, 52.6%, and 1.1 months, respectively. The main grade 3 and 4 toxicities were leukocytopenia (35%), neutropenia (65%), thrombocytopenia (30%), and elevations of the aspartate aminotransferase (45%) and alanine aminotransferase (35%) levels. These toxicities were generally brief and reversible. Conclusion Transcatheter arterial infusion chemotherapy with an epirubicin-Lipiodol emulsion appears to have only modest activity with moderate toxicity for treatment of patients with TAE-refractory HCC. These findings do not support its use in practice, and further studies with the same regimen in patients with TAE-refractory HCC are not recommended.