Oral smoothened inhibitors for Gorlin syndrome: A clinical review.

Authors :: Baczynski A
Cahn B
Worley B
Haber R
Alam M
Source :: Journal of the American Academy of Dermatology [J Am Acad Dermatol] 2024 Oct; Vol. 91 (4), pp. 706-711. Date of Electronic Publication: 2024 Jun 29.
Publication Year :: 2024
Abstract: Although smoothened inhibitors (SMOi) have demonstrated efficacy in the management of basal cell carcinoma, no guidelines are available on how to utilize SMOi in the treatment of Gorlin syndrome (GS). This review's objective is to assess the clinical response to SMOi in GS, provide practical guidance for clinicians, and identify areas for future research. Through comprehensive searches of previous publications and expert opinion, this review demonstrates that intermittent dosing of SMOi and daily dosing have similar efficacy. While the adverse events of SMOi may result in their discontinuation during treatment of GS, intermittent dosing may improve compliance.<br />Competing Interests: Conflicts of interest None disclosed.<br /> (Copyright © 2024 American Academy of Dermatology, Inc. Published by Elsevier Inc. All rights reserved.)