Axitinib plus best supportive care resulted in significantly longer progression-free survival and time to tumor progression and higher clinical benefit rate than placebo plus best supportive care in patients with advanced hepatocellular carcinoma (HCC), a new study published online ahead of print in the journal Annals of Oncology has shown.1

For the phase 2 study, researchers sought to assess the efficacy and safety of axitinib, a potent and selective vascular endothelial growth factor receptors 1-3 inhibitor, plus best supportive care in patients with locally advanced or metastatic HCC.

Researchers enrolled patients with HCC and Child-Pugh Class A who progressed on or were intolerant to 1 prior antiangiogenic therapy and randomly assigned them 2:1 to receive axitinib twice daily combined with best supportive care or placebo plus best supportive care.

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Results showed no significant difference in overall survival, the primary endpoint, between axitinib and placebo; however, improvements favoring axitinib plus best supportive care were observed in progression-free survival, time to tumor progression, and clinical benefit rate.

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Researchers found that there was no significant difference in overall response rate between the 2 treatment arms and patient-reported outcomes favored placebo plus best supportive care.

In regard to safety, the most common adverse events in the axitinib group were diarrhea, hypertension, and decreased appetite.


  1. Kang Y-K, Yau T, Park J-W, et al. Randomised phase II study of axitinib versus placebo plus best supportive care in second-line treatment of advanced hepatocellular carcinoma [published online ahead of print September 18, 2015]. Ann Oncol. doi: 10.1093/annonc/mdv388.