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Can Vismodegib Therapy Improve Visual Function for Patients With Basal Cell Carcinoma?

By: Joshua D. Madera, MS
Posted: Thursday, August 26, 2021

For patients with orbital and extensive periocular basal cell carcinoma, treatment with the Hedgehog pathway inhibitor vismodegib may preserve eye and visual function, according to the VISORB trial published in The Oncologist. The findings were consistent regardless of whether vismodegib was used as primary or neoadjuvant treatment, explained Francis P. Worden, MD, of the Rogel Comprehensive Cancer Center, Ann Arbor, Michigan, and colleagues.

From 2015 to 2019, a total of 34 patients with globe and lacrimal drainage system that threatened orbital and extensive periocular basal cell carcinoma were recruited for the open-label, nonrandomized phase IV clinical trial. Patients received 150 mg of vismodegib daily for up to 12 months. The Visual Assessment Weighted Score (VAWS) and standard ophthalmic exams were used to assess patients’ visual function. In addition, surgical excision samples were analyzed histologically by dermatopathologists.

The study authors reported a mean VAWS of 44/50, 46/50, and 47/50 at baseline, 3 months, and 12 months, respectively. In addition, as compared with baseline, a total of 79.4% of patients maintained a stable score or demonstrated improvement. In contrast, 3% of patients experienced a score decline of at least 5 points, and 14.7% of patients experienced a score decline of 2 to 4 points. Furthermore, 47% of patients had complete tumor regression, as identified by MRI, and 56% of patients had complete tumor regression, as identified by physical examination. Moreover, of the 27 patients who underwent surgical excision, 67% showed no evidence of disease, 22% had residual disease with clear margins, and 11% demonstrated residual disease extending to margins.

“Vismodegib may be most useful as a neoadjuvant therapy, with a 3- to 6-month course of medical therapy preceding surgical excision for histologic clearance and potential cure. Such an approach would maximize utility of the drug for organ preservation while minimizing toxicity and adverse events,” suggested Dr. Worden.

Disclosure: For full disclosures of the study authors, visit onlinelibrary.wiley.com.



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