Treating periocular basal-cell carcinoma with vismodegib before surgery may reduce the size of the tumor, make the surgery less disfiguring and help preserve the eye, according to a single-center study

"Observations in this report confirm our hypothesis that availability of sonic hedgehog inhibitors such as vismodegib have improved the outcomes in patients with locally advanced recurrent orbital and periocular basal-cell carcinoma and have led to a decreased need for mutilating organ-sacrificing surgery such as orbital exenteration," Dr. Bita Esmaeli told Reuters Health by email.

Dr. Esmaeli and her colleagues at the University of Texas MD Anderson Cancer Center in Houston examined the records for all patients with T4 periocular basal-cell carcinoma she had treated with neoadjuvant vismodegib prior to surgery between 2013 and 2017.


All eight patients were men between 66 and 84 years of age, and six had recurrent disease, the team reports in the British Journal of Ophthalmology, online July 18. Vismodegib was used to treat one patient with an unresectable tumor, six patients to avoid orbital exenteration and one patient to avoid disfiguring facial surgery.

After four to 36 (median 14) months of treatment with vismodegib followed by eye-sparing surgery, all final surgical margins were negative for tumor. No microscopic residual basal-cell carcinoma was found during surgery in five patients; three had the significant partial response with residual tumor detected on pathology.

Co-author Dr. Bonnie S. Glisson told Reuters Health by email, "The eye-sparing approach that was pioneered by Dr. Esmaeli demonstrates that vismodegib is a well-tolerated oral molecularly targeted drug with high effectiveness, safety, and feasibility in this setting."

"Vismodegib or other sonic hedgehog inhibitors should be considered for patients with locally advanced or recurrent orbital and periocular basal cell carcinoma who would otherwise be headed for an orbital exenteration," Dr. Esmaeli noted.

"Experience with mitigating the low-grade side effects of the drug, the duration of treatment prior to surgical intervention, and mitigating the cost and insurance approval are all important considerations for this approach."

Dr. Hassan Shah, an assistant professor of ophthalmology and visual science at the University of Chicago Medical Center, said, "This study is important because it describes an additional method of treating facial basal-cell carcinoma that can help avoid significant reconstructive surgery.

In specific situations, this method of treatment may be the best option for a patient, and it helps to improve our ability to tailor individual treatment regimens."

"It is important to note that the findings of this treatment can only be applied to very specific situations, not to everyone with basal-cell carcinoma," Dr. Shah, who was not involved in the study, told Reuters Health by email. "This may be the best option for some of our patients (especially older people) who may not be able to tolerate many months of medical therapy."