Efficacy and safety of axitinib in patients with advanced non-small-cell lung cancer: Results from a phase II study

Joan H. Schiller, Timothy Larson, S. H Ignatius Ou, Steven Limentani, Alan Sandler, Everett Vokes, Sinil Kim, Katherine Liau, Paul Bycott, Anthony J. Olszanski, Joachim Von Pawel

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150 Scopus citations


Purpose: This phase II study evaluated efficacy and safety of single-agent axitinib, an oral, potent, selective inhibitor of vascular endothelial growth factor receptors (VEGFR) -1, -2, and -3, in patients with advanced non-small-cell lung cancer (NSCLC). Patients and Methods: This was an open-label, single-arm, multicenter, phase II study with a Simon two-stage minimax design. Patients received a starting dose of axitinib 5 mg orally BID. The primary end point was Response Evaluation Criteria in Solid Tumors (RECIST) -defined objective response rate. Secondary end points included safety and tolerability, overall survival (OS), and progression-free survival (PFS). Results: Thirty-two patients were enrolled, with a median age of 66.5 years. The majority of patients (75%) had adenocarcinoma. Nine patients (28%) had received no prior chemotherapy for metastatic disease, and 23 (72%) had received ≥ one regimen. Three patients (9%) had a RECIST investigator-assessed, confirmed partial response (PR); disease control rate (PR + stable disease) was 41%. Median PFS was 4.9 months overall (95% CI, 3.6 to 7.0 months). Median OS was 14.8 months (95% CI, 10.7 months to not estimable) overall and 14.8 months (95% CI, 12.5 months to not estimable) in patients receiving first-line axitinib. One-year survival rates for patients with or without prior therapy for metastatic disease were 57% and 78%, respectively. Grade 3 treatment-related adverse events in ≥ 5% of patients comprised fatigue (22%), hypertension (9%), and hyponatremia (9%). Conclusion: Axitinib demonstrated single-agent activity in patients with advanced NSCLC. Therapy was well tolerated with manageable toxicities. Further investigation of this VEGFR inhibitor in NSCLC is of interest.

Original languageEnglish (US)
Pages (from-to)3836-3841
Number of pages6
JournalJournal of Clinical Oncology
Issue number23
Publication statusPublished - Aug 10 2009


ASJC Scopus subject areas

  • Cancer Research
  • Oncology
  • Medicine(all)

Cite this

Schiller, J. H., Larson, T., Ou, S. H. I., Limentani, S., Sandler, A., Vokes, E., ... Von Pawel, J. (2009). Efficacy and safety of axitinib in patients with advanced non-small-cell lung cancer: Results from a phase II study. Journal of Clinical Oncology, 27(23), 3836-3841. https://doi.org/10.1200/JCO.2008.20.8355