The oral targeted agent sorafenib significantly prolongs survival in patients with advanced hepatocellular carcinoma compared with placebo, according to results of a phase III trial presented at the annual meeting of the American Society of Clinical Oncology.

“This is the first time we’ve had an effective systemic treatment for liver cancer,” said Joseph Llovet, MD, Director of research in liver cancer at Mount Sinai School of Medicine, New York City, and lead author of the study. “Our findings demonstrated survival advantages that are both statistically significant and clinically meaningful.”

In the SHARP trial, researchers examined overall survival and time to progression in patients who had previously untreated liver cancer who were randomized to 400 mg sorafenib twice daily (299 patients) or placebo (303 patients) for six months.

Patients randomized to sorafenib versus placebo survived 44 percent longer, with a median survival of 10.7 months compared with 7.9 months for patients in the placebo group. Time to progression was also longer in the sorafenib group: 5.5 months versus 2.8 months. The magnitude of advantage with active treatment was so large the trial was ended early.

Incidence of side effects was similar between groups (52 percent, sorafenib, versus 54 percent, placebo). The most common moderate to severe side effects were diarrhea (11 percent versus 2 percent), skin reactions in hands and feet (8 percent versus 1 percent), fatigue (10 percent versus 15 percent), and bleeding (6 percent versus 9 percent).