A phase II trial of second-line axitinib following prior antiangiogenic therapy in advanced hepatocellular carcinoma.

Mairead Mcnamara, Lisa W Le, Anne M Horgan, Alex Aspinall, Kelly W Burak, Neesha Dhani, Eric Chen, Mehrdad Sinaei, Glen Lo, Tae Kyoung Kim, Patrik Rogalla, Oliver F Bathe, Jennifer J Knox

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    BACKGROUND: Second-line treatment options in advanced hepatocellular carcinoma (HCC) are limited. Axitinib, a selective potent tyrosine kinase inhibitor (TKI) of vascular endothelial growth factor VEGF) receptors 1, 2, and 3, merits exploration in HCC. METHODS: This was a single-arm phase II trial of axitinib in advanced HCC. Eligible patients were Child-Pugh A/B7, with measurable progressive disease after TKIs/antiangiogenic drugs. Axitinib was started at 5 mg twice daily orally, titrated from 2 to 10 mg twice daily as tolerated. The primary end point was tumor control at 16 weeks by RECIST1.1; secondary end points were response rate, comparing response by RECIST1.1 to Choi and modified RECIST, exploring dynamic contrast-enhanced imaging models, safety, progression-free (PFS), and overall survival (OS). RESULTS: Thirty patients were treated. Of 26 patients evaluable for response, there were 3 partial responses (PR) per RECIST1.1; 13 PR by Choi, 6 PR and 1 complete response by modified RECIST. Tumor control rate at 16 weeks was 42.3%. Two-week perfusion changes were noted on functional imaging. Of 21 patients with evaluable α-fetoprotein response, 43% had >50% decrease from baseline. Most common axitinib-related grade 3/4 adverse events (AEs) were hypertension, thrombocytopenia and diarrhea. Of 11 patients with any grade hypertension, 7 had disease control >36wks. Four patients discontinued treatment due to AEs. Median PFS was 3.6months. Median OS was 7.1months. CONCLUSIONS: With 42.3% tumor control at 16weeks, primary endpoint was met. Axitinib has shown encouraging tolerable clinical activity in VEGF-pretreated HCC patients but further study should be in a selected population incorporating potential biomarkers of response. Cancer 2015. © 2015 American Cancer Society.
    Original languageEnglish
    Publication statusPublished - 6 Jan 2015


    • HCC
    • axitinib
    • phase II trial
    • prior antiangiogenic therapy
    • second-line treatment


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