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Original Investigation
Clinical Sciences
December 2013

Vismodegib for Periocular and Orbital Basal Cell Carcinoma

Author Affiliations
  • 1Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, Ontario, Canada
  • 2Division of Oculofacial Plastic and Orbital Surgery, California Pacific Medical Center, San Francisco, California
  • 3Department of Dermatology, Stanford University School of Medicine, Palo Alto, California
  • 4Division of Dermatology and Dermatologic Surgery, Scripps Clinic, La Jolla, California
JAMA Ophthalmol. 2013;131(12):1591-1594. doi:10.1001/jamaophthalmol.2013.5018
Abstract

Importance  Basal cell carcinoma (BCC) represents 90% of malignant eyelid tumors and is locally invasive and destructive, if left untreated.

Objective  To assess the feasibility of using vismodegib for periocular and orbital BCC based on its efficacy and tolerability.

Design, Setting, and Participants  In this prospective observational case series, consecutive patients with periocular or orbital BCC who met criteria for treatment with vismodegib were recruited prospectively during an 8-month period from February through September 2012 from 2 academic hospitals. Seven patients received oral vismodegib, 150 mg daily, until maximum clinical response was achieved, the tumor progressed, or the patient could no longer tolerate adverse effects. Clinical response and adverse effects related to treatment were recorded. The primary endpoint was reduction in lesion size, measured as percentage change in the externally visible dimension.

Exposure  Oral vismodegib.

Results  All 7 patients had locally advanced, biopsy-proven, infiltrative BCC that was not amenable to surgical resection or radiation. No patients had metastatic disease at presentation. The mean patient age was 71 years (range, 43-100 years), and 4 patients (57%) had secondary orbital involvement. The mean lesion size was 3.4 cm (range, 1.0-6.0 cm), and all 7 cases (100%) represented recurrent tumors excised previously with controlled margins by frozen section or Mohs micrographic surgery. The mean treatment duration was 11 weeks (range, 4-16 weeks), and the mean duration of follow-up was 7.3 months (range, 5-10 months). Two patients (29%) demonstrated complete clinical regression, 2 (29%) demonstrated greater than 80% partial clinical regression, 2 (29%) demonstrated less than 35% partial clinical regression, and 1 (14%) progressed. Adverse reactions occurred in 6 patients (86%) and included alopecia (29%), dysgeusia (29%), muscle cramps (29%), and anorexia (14%). Two patients (29%) developed new squamous cell carcinomas (well-differentiated, keratoacanthoma type) at uninvolved sites including the eyebrow and forearm.

Conclusions and Relevance  Vismodegib seems to be well-tolerated and effective for treating periocular and orbital BCC in about half of all cases. Patients receiving treatment should be monitored for new squamous cell carcinomas at uninvolved sites.

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