Transarterial infusion chemotherapy alone versus transarterial chemoembolization for the treatment of hepatocellular carcinoma: Results of a multicenter randomized phase III trial

Abstract

4643 Background: Transarterial chemoembolization (TAE) is widely used for patients (pts) with hepatocellular carcinoma (HCC) although evidence of a survival benefit remains controversial. A multicenter phase III trial was designed to compare the effects of TAE and transarterial infusion chemotherapy alone (TAI) on overall survival. Methods: Pts with newly diagnosed unresectable HCC were randomly assigned to either the TAE or the TAI group, stratified according to center and the serum alpha-fetoprotein level. Zinostatin stimalamer (SMANCS), up to a maximum of 6 mg, plus lipiodol emulsion with or without gelatin sponge was injected into the hepatic artery. Treatment was repeated when a follow-up computed tomography examination showed the appearance of new lesions in the liver or the regrowth of the treated tumors. Based on our previous phase II studies, reporting a 2-year survival rate of 80% in pts treated with TAE and of 60% in pts treated with TAI, 80 pts were needed in each group to achieve a level of statistical significance of 5% (2-sided) and a power of 80%. Results: From October 1999 to June 2003, 79 pts were assigned to the TAE group and 82 pts to the TAI group. The two groups were well balanced with regard to their baseline characteristics. The total number of treatment courses was 170 with a mean of 2.2 courses per patient (range, 1–9 courses) in the TAE group and 193 with a mean of 2.4 courses (range, 1–6 courses) in the TAI group. As of June 2005, 51 pts in the TAE group and 58 pts in the TAI group had died. The median overall survival time was 646 days in the TAE group and 679 days in the TAI group. No significant difference in survival was seen between the two groups (p=0.383). The mortality rate reduction for overall survival was 15.8% (95% CI, -24.1% to 42.9%) for the TAE relative to the TAI alone. Conclusions: This study suggests that treatment intensification as a result of embolization did not increase survival, compared with SMANCS transarterial chemotherapy alone, in pts with HCC. No significant financial relationships to disclose.