There is no effective systemic therapy for patients with hepatocellular carcinoma. A recent trial reported a moderate antitumor activity for gemcitabine among Asian patients with advanced hepatocellular carcinoma. This led to our examination of the efficacy and tolerability of the drug in a population of U.S. patients.

Thirty patients with measurable, unresectable, or metastatic hepatocellular carcinoma who had received at least one previous form of systemic therapy were enrolled. All patients were required to have adequate major organ function and performance status. Patients received gemcitabine (1000 mg/m² intravenously over 30 minutes weekly) for 3 consecutive weeks followed by a 1‐week rest. Patients were assessed radiographically every 8 weeks.

All 30 patients were evaluable for response and toxicity. Ninety cycles of therapy were administered (median 2, range 1–8). No complete or partial responses were observed. Nine patients (30%) had stable disease (median duration 7.4 months, range 2–17). Median survival for all 30 patients was 6.9 months (95% confidence interval, 4.5–13.5) and the 1‐year survival rate was 40%. Mild hematologic toxicity occurred. Two patients (7%) developed Grade 4 neutropenia and one patient (3%) experienced Grade 3 thrombocytopenia. There were no episodes of febrile neutropenia. One patient who had previously undergone orthotopic liver transplantation developed hemolytic‐uremic syndrome that resolved with discontinuation of chemotherapy and plasmapheresis.

Although generally well tolerated, gemcitabine had minimal effect in patients with advanced hepatocellular carcinoma. Cancer 2002;94:3186–91. © 2002 American Cancer Society.

DOI 10.1002/cncr.10607