Objectives: A prospective randomized study was performed to test the hypothesis that tamoxifen might improve the survival of patients with advanced hepatocellular carcinoma (HCC) and to correlate the response of treatment with the expression of hormone receptors.
Methods: One hundred nineteen patients with advanced and otherwise untreatable HCC were included in a placebo-controlled, single-blind trial. The patients were randomized to tamoxifen group (61 patients) and control group (58 patients) and were prescribed with a daily dose of 30 mg of tamoxifen and placebo, respectively. Immunohistochemical tests for estrogen and progesterone receptors were performed on the tumor tissues obtained from 66 patients. All patients were closely monitored and the survival outcome of the two groups of patients was compared and stratified according to the hormonal receptor status.
Results: There was no difference in the 1-month mortality rates (32.8% vs. 43.1%, p = 0.246) and the median survival (44 days vs. 41 days, p = 0.703) between the tamoxifen group and the control group. Furthermore, the expression of hormone receptors in the tumors did not affect the survival outcome of the patients treated with tamoxifen. None of the patients who survived longer than 3 months had tumor that had partial response to tamoxifen treatment on follow-up imaging study.
Conclusions: Tamoxifen has no efficacy in the treatment of patients with advanced HCC and response to treatment was not affected by the expression of hormone receptors.