Controlled clinical trial of doxorubicin and tamoxifen versus tamoxifen monotherapy in hepatocellular carcinoma

Objectives Treatment results of advanced hepatocellular carcinoma have rema ined unsatisfactory; the response rates to intravenous doxorubicin are no b etter than 20%. Oral tamoxifen has been proposed on the basis of beneficial results in some trials. The aim of this study was to evaluate whether the addition of doxorubicin to oral tamoxifen improves survival compared to ora l tamoxifen alone. Methods Thirty-two consecutive patients with a priori defined contra-indica tions against surgery (transplantation, resection) or chemo-embolization we re evaluated to receive chemotherapy. All patients received oral tamoxifen 30 mg bid; 16 also received intravenous doxorubicin 50 mg/m(2) every 4 week s. The control group consisted of the remaining 16 patients who either were considered unfit for doxorubicin because of a Karnofsky index < 50% (n = 5 ), cardiac disease (n = 6) or who refused to have cytotoxic drug therapy (n = 5), Results Median survival time was 148 days (95% CI 89.2-206.8) in the doxoru bicin group and 96 days (95% CI 49.0-143.0) in the control group, and this was not significantly different (P = 0.408), regardless of the presence or absence of cirrhosis. Conclusions In conclusion, the results of our study indicate that combinati on therapy using doxorubicin and tamoxifen is unlikely to considerably impr ove survival compared to tamoxifen alone in patients with advanced hepatoce llular carcinoma. Eur J Gastroenterol Hepatol 12:281 -284 (C) 2000 Lippinco tt Williams & Wilkins.