• Aleksandar Sekulic, Michael R Migden, Karl Lewis, John D Hainsworth, James A Solomon, Simon Yoo, Sarah T Arron, Philip A Friedlander, Ellen Marmur, Charles M Rudin, Anne Lynn S Chang, Luc Dirix, Jeannie Hou, Huibin Yue, Axel Hauschild, and ERIVANCE BCC investigators.
• Mayo Clinic, Scottsdale, Arizona. Electronic address: sekulic.aleksandar@mayo.edu.
• J. Am. Acad. Dermatol. 2015 Jun 1;72(6):1021-6.e8.

BackgroundPrimary analysis from the pivotal ERIVANCE BCC study resulted in approval of vismodegib, a Hedgehog pathway inhibitor indicated for treatment of adults with metastatic or locally advanced basal cell carcinoma (BCC) that has recurred after surgery or for patients who are not candidates for surgery or radiation.ObjectiveAn efficacy and safety analysis was conducted 12 months after primary analysis.MethodsThis was a multinational, multicenter, nonrandomized, 2-cohort study in patients with measurable and histologically confirmed locally advanced or metastatic BCC taking oral vismodegib (150 mg/d). Primary outcome measure was objective response rate (complete and partial responses) assessed by independent review facility.ResultsAfter 12 months of additional follow-up, median duration of exposure to vismodegib was 12.9 months. Objective response rate increased from 30.3% to 33.3% in patients with metastatic disease, and from 42.9% to 47.6% in patients with the locally advanced form. Median duration of response in patients with locally advanced BCC increased from 7.6 to 9.5 months. No new safety signals emerged with extended treatment duration.LimitationsLimitations include low prevalence of advanced BCC and challenges of designing a study with heterogenous manifestations.ConclusionThe 12-month update of the study confirms the efficacy and safety of vismodegib in management of advanced BCC.Copyright © 2015 American Academy of Dermatology, Inc. Published by Elsevier Inc. All rights reserved.

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