TREATMENT OF HEPATOCELLULAR-CARCINOMA WITH TAMOXIFEN - A DOUBLE-BLIND PLACEBO-CONTROLLED TRIAL IN 120 PATIENTS

Background & Aims: The progression of hepatocellular carcinoma may be influenced by estrogens. This has offered the rationale for evaluating the therapeutic usefulness of estrogen-receptor blockers; it is being debated whether long-term tamoxifen administration improves survival in patients with this neoplasm. The aim of this study was to assess the efficacy of tamoxifen administration in the treatment of hepatocellular carcinoma. Methods: One hundred twenty patients with this neoplasm who were not suitable for surgery, ethanol injection, or transarterial embolization were included in a placebo-controlled trial and randomized to tamoxifen, 20 mg/day per os, (group A, n = 58) or placebo (group B, n = 62), Patients with terminal diseases were excluded, Results: Both groups were similar with regard to sex, age, liver function (Child-Pugh's score, 6.5 +/- 1.4 vs. 6.4 +/- 1.4), baseline performance status, and tumor stage, Tamoxifen had no antitumoral effect with no differences in the survival between groups (1- and a-year actuarial rate: group A, 51% and 27%; and group B, 43% and 29%; P = 0.75), even when stratifying patients according to baseline status, Furthermore, there were no differences in the probability of disease progression (P = 0.46) and baseline performance status maintenance (P = 0.93) during follow-up, Conclusions: Tamoxifen has no efficacy in the treatment of patients with advanced hepatocellular carcinoma.