Phase II Study to Topotecan and Cisplatin in Advanced Hepatocellular Carcinoma

BACKGROUND: Hepatocellular carcinoma remains a highly chemoresistant neoplasm and is a common malignancy with poor prognosis in Korea. We performed a phase II study to evaluate the efficacy and toxicities of topotecan and cisplatin combination chemotherapy for advanced hepatocellular carcinoma. METHODS: Between November 1999 and May 2001, ten patients with histologically proven hepatocellular carcinoma were enrolled in this study. The median age was 54 (range: 53–74) years and all were male. Six patients demonstrated stage IV, 1 stage IIIC, 2 stage IIIB and 1 stage IIIA. Six patients showed a ECOG performance status of 1. The treatment regimen consisted of topotecan 1.25 mg/m(2) and cisplatin 20 mg/m(2) for 5 days. The treatment was repeated every 4 weeks. Toxicities were evaluated according to WHO toxicity criteria. RESULTS: All ten patients were evaluable for response and toxicity. There was only one patient who achieved partial response. The overall response rate was 10% (95% C.I.) and the response duration was 46 weeks. The median survival of all patients was 21 (range: 17–54+) weeks. During a total of 24 cycles, neutropenia of WHO grade 3 and 4 occurred in 33%, thrombocytopenia in 33% and anemia in 21%. In non-hematologic toxicity, diarrhea and hepatoxicity of grade 3 occurred in 1 and 2 patients, respectively. But there was no treatment-related death. CONCLUSION: When used in this dose and schedule, topotecan and cisplatin combination chemotherapy does not seem to be effective for patients with advanced hepatocellular carcinoma.