A phase II study of pegylated liposomal doxorubicin for treatment of advanced hepatocellular carcinoma

Background: Pegylated liposomal doxorubicin has an enhanced efficacy and re duced toxicity compared with free doxorubicin. The efficacy and toxicity of pegylated liposomal doxorubicin was investigated in patients with hepatoce llular carcinoma. Patients and methods: Patients with histologically confirmed, locally advan ced or metastatic hepatocellular carcinoma and a Karnofsky index > 60% were included in this prospective single-arm study. Exclusion criteria were liv er cirrhosis stage Child-Pugh C, previous chemotherapy, or chemoembolizatio n. Pegylated liposomal doxorubicin was given in a dose of 30 mg/m(2) every three weeks until progression of disease. After inclusion of five patients the dose could be escalated to 40 mg/m(2) in absence of toxicity grade 3 an d 4. Results: Sixteen patients were evaluable for response. No objective respons e was achieved. The median survival time was 140 days (95% confidence inter val: 126-154 days). Treatment toxicities grade greater than or equal to 3 c omprised increased liver enzymes in patients with preexisting grade 1 or 2 elevation (n = 6), hematologic toxicity (n = 5), and hypersensitivity (n = 2). Conclusions: Pegylated liposomal doxorubicin is not effective for treatment of advanced hepatocellular carcinoma. The favorable toxicity profile was c onfirmed even in patients with underlying liver disease.