The VEGF inhibitor axitinib has limited effectiveness as a therapy for adrenocortical cancer

Context: Adrenocortical carcinoma (ACC) is a rare malignancy with a poor prognosis in need of more effective treatment options. Published evidence indicates many ACCs express the vascular endothelial growth factor receptor (VEGFR), suggesting inhibiting vascular endothelial growth factor signaling could potentially impact tumor growth. Objective: The objective of the study was to determine the antitumor efficacy of axitinib (AG-013736), a potent, selective inhibitor of VEGFR1, -2, and -3. Design: This was a phase II, open-label trial using a two-stage design. Patients: Thirteen patients with metastatic ACC previously treated with at least one chemotherapy regimen with or without mitotane participated in the study. Intervention: Starting axitinib dose was 5 mg orally twice daily. Dose escalations were permitted if the administered dose was tolerable. Results: Thirteen patients were enrolled. Dose escalation was possible in seven patients, but the majority could not tolerate a dose higher than the starting ...