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Vismodegib Discontinuation and Survival in Locally Advanced Basal Cell Carcinoma

By: Cordi Craig
Posted: Thursday, November 7, 2019

Patients with locally advanced basal cell carcinoma who receive the Hedgehog pathway inhibitor vismodegib often experience treatment-related adverse effects that result in drug discontinuation. However, a retrospective study published in the Journal of Clinical Oncology found that after discontinuing treatment, patients frequently maintained long-term responses after complete remission. Florian Herms, MD, of the Université Paris Diderot, Paris, and colleagues also found that even after relapse, responses appear to be achieved with vismodegib retreatment.

The investigators evaluated relapse-free survival in 116 patients with locally advanced basal cell carcinoma from nine French oncodermatology units. All patients achieved complete responses with vismodegib and discontinued treatment between March 2012 and January 2016.

The median relapse-free survival was 18.4 months. The median overall survival was not reached, but at 36 months, the relapse-free survival rate was 35.4% for the total population and 40% for patients without Gorlin syndrome (also known as nevoid basal cell carcinoma syndrome, Gorlin syndrome is a rare condition that may increase the risk of developing various malignant and benign tumors). The localization of disease to the limbs and trunk was the only variable that was independently associated with a higher risk of relapse (P = .019).

At 36 months, the cumulative incidence of relapse was 59%. Relapse was focal in 35 patients and multifocal in 19 patients. Of the patients who relapsed, 27 were re-treated with vismodegib, and 23 (85%) resulted in an objective response. The complete and partial response rates were 37% and 48%, respectively. Of the patients who relapsed during follow-up, 24 were eligible for surgery.

“Long-term response after vismodegib discontinuation is frequent,” the investigators concluded. “Most patients who experience a relapse still respond to vismodegib rechallenge.”

Disclosure: The full disclosures of the study authors can be found at

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