FROM THE LITERATURE
HEPATOCELLULAR CARCINOMA:
Improved Survival Seen In Advanced Disease with Sorafenib

The incidence of HCC is increasing in the U.S. and Europe, and it is the third highest cause of cancer-related death globally, behind only lung and stomach cancers. No systemic therapy has improved survival in patients with advanced HCC. A preliminary study suggested that sorafenib (Nexavar), an oral multikinase inhibitor of the vascular endothelial growth factor receptor (VEGFR), the platelet-derived growth factor receptor β (PDGFR-β), and Raf may be effective in HCC. Results of this uncontrolled phase II trial of a single-agent sorafenib demonstrated a median OS of 9.2 months and a median time to progression (TTP) of 5.5 months.

On the basis of these data, investigators conducted a large (602 patient) phase III, randomized, double blind, placebo-controlled trial to assess the efficacy and safety of sorafenib in patients with advanced HCC who had not received previous systemic treatment. Patients received either sorafenib 400 mg PO twice daily or placebo. Primary outcomes were OS and the time to symptomatic progression. Secondary outcomes were time to radiologic progression and safety.

At the second planned interim analysis, 321 deaths had occurred, and the study was stopped. Median OS was 10.7 months in the sorafenib group and 7.9 months in placebo patients (HR in sorafenib group, 0.69; p<0.001). There was no significant difference between the two groups in the median time to symptomatic progression (4.1 months vs. 4.9 months; p=0.77). The median time to radiologic progression was 5.5 months in the sorafenib group and 2.8 months in the placebo group (p<0.001). Seven patients in the sorafenib group (2%) and two patients in the placebo group (1%) had a partial response; no patients had a complete response. Diarrhea, weight loss, hand-foot skin reaction, and hypophosphatemia were more frequent in the sorafenib group.

Conclusions/Summary:

  1. This study showed that sorafenib prolonged median survival and the time to progression by nearly 3 months in patients with advanced HCC.
  2. Future studies are warranted to evaluate sorafenib as an adjuvant therapy after curative or locoregional therapies.
  3. Studies are needed to evaluate sorafenib in combination with other molecular targeted therapies.

Llovet JM, Ricci S, Mazzaferro V, et al. Sorafenib in Advanced Hepatocellular Carcinoma.
N Engl J Med. 2008;378-390.

We welcome your comments and suggestions regarding content and format at primelines@primeoncology.org.

    prime oncology logo
plines footer