Epirubicin and etoposide combination chemotherapy to treat hepatocellular carcinoma patients: a phase II study.

Approximately half the patients affected with hepatocellular carcinoma (HCC) present with unresectable disease, so that efficacious systemic chemotherapy protocols are badly needed. We report the results of a phase-II study aimed at testing the efficacy and toxicity of a combination of epirubicin and VP-16. Thirty six patients (80 men and 6 women) received epirubicin (40 mg/m2, on day 1) and VP-16 (120 mg/m2, on days 1, 3 and 5) every 28th day. Chemotherapy was stopped in case of disease progression, while the patients who achieved an objective response or who had stable disease continued treatment for a maximum of 10 cycles. One patient (3%) achieved a complete response, while 13 patients (36%) achieved partial response, i.e. 14 objective responses in all (39%, 95% CI: 23-55%). 11 patients (31%) exhibited stable disease, while in the other 11 patients (31%) the disease progressed. Median overall survival time was 10 months and 13.5 months in the subgroup of patients responding to treatment. Significant, especially haematological, toxicity was documented, but in no case was it so severe as to require discontinuation of treatment or reduction of the dosage. In conclusion, this combination appears to be an active and tolerable therapeutic option for HCC patients who are not candidates for surgical or locoregional procedures, and in our opinion it deserves further exploration within a randomised controlled trial versus best supportive therapy.