Cheng, A. L. et al. Efficacy and safety of sorafenib in patients in the Asia-Pacific region with advanced hepatocellular carcinoma: a phase III randomised, double-blind, placebo-controlled trial. Lancet Oncol. 10, 25–34 (2009).

Surgical resection is not a treatment option for 80% of patients who present with intermediate or advanced hepatocellular carcinoma. Sorafenib—an oral multikinase inhibitor with antiangiogenic and antiproliferative effects—notably improves overall survival in Asian-Pacific patients with advanced hepatocellular carcinoma, according to a new study. Previous studies have shown that sorafenib is efficacious and well-tolerated in a similar group of patients from North America and Europe. The prevalence of advanced hepatocellular carcinoma is, however, greatest in the Asia-Pacific region (75% of all cases globally), with different underlying etiologies, prompting Cheng and colleagues to study the efficacy and safety of sorafenib in this group of patients.

Patients with hepatocellular carcinoma from South Korea, China, and Taiwan were randomly assigned to receive either oral sorafenib (400 mg, n = 150) or placebo (n = 76) twice-daily in 6-week cycles. Tumor measurements were made at baseline and at the end of each treatment cycle; safety and drug accountability monitoring and symptom progression were assessed every 3 weeks.

Patients treated with sorafenib had a significantly higher median overall survival compared with those in the placebo group (6.5 months versus 4.2 months). Time to progression was also increased with sorafenib therapy (2.8 months versus 1.4 months); time to symptomatic progression was similar in both groups. Sorafenib was generally well-tolerated with manageable adverse effects.

Cheng is excited by the success of sorafenib treatment of advanced hepatocellular carcinoma, “We are now working on the combinations of sorafenib with other targeted agents, as well as combinations of sorafenib with metronomic chemotherapy,” he reveals.