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  • Vismodegib for Basal Cell Carcinoma

    By Lynda Seminara
    Selected By: Richard K. Parrish II, MD
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    Journal Highlights

    American Journal of Ophthalmology, November 2019

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    Basal cell carcinoma (BCC), the most common skin cancer, accounts for 90% of malignant tumors of the eyelid. Eiger-Moscovich et al. looked at the effectiveness of vismodegib, a Hedge­hog pathway inhibitor, for treating orbital and advanced periocular BCC. They found that treatment according to an individualized maximally tolerated dose achieved responses similar to those achieved in the pivotal ERIV­ANCE study. The authors emphasized that longer-term studies are needed to gauge prognosis.

    This retrospective series included 21 patients (median age, 76 years; 16 men) with biopsy-proven periocular BCC (n = 6) or orbital BCC (n = 15). In most cases, treatment was given for five to seven months, at the usual dosage of 150 mg per day, followed by an intermission. If deterioration was observed, treatment was resumed. The aim was to customize each patient’s treatment to the maximally tolerated dose. Some patients received a partial dose to minimize adverse events such as hepatotoxicity.

    The median duration of vismodegib treatment was nine months. The me­dian follow-up period was 17 months after treatment cessation. The clinical response was complete in 10 patients, partial in 10 others, and stable in one patient. No patient had progressive disease (defined as an increase in tumor size of >20%). Among the complete responders, two were still being treated and eight had finished treatment at the time of this report. Five of the eight maintained their complete response by 16 months; the other three had recur­rence within eight months.

    Treatment response did not seem affected by orbital involvement or tumor stage. Nearly all treatment-related adverse reactions were low grade; the most common were muscle spasm (76%) and dysgeusia (57%). The only grade 3/4 adverse event was hepato­toxicity (10%). Eight patients discon­tinued treatment due to side effects. Five patients died, most from reasons that appeared unrelated to vismodegib. However, one death (from sepsis) may have been related to treatment.

    To the authors’ knowledge, this is the largest study of vismodegib therapy for locally advanced periocular BCC. Response rates to maximally tolerated doses were comparable to those with the ERIVANCE protocol, yet the op­timal treatment protocol remains un­known, and longer studies are needed.

    The original article can be found here.