Oral smoothened inhibitors for Gorlin Syndrome: A clinical review

Affiliations

Advocate Illinois Masonic Medical Center

Abstract

Background:Although smoothened inhibitors (SMOi) have demonstrated efficacy in the management of basal cell carcinoma (BCC), no guidelines are available on how to utilize SMOi in the treatment of Gorlin syndrome (GS).

Objective:Assess the clinical response to SMOi in GS, provide practical guidance for clinicians, and identify areas for future research.

Methods:Alongside a comprehensive search of Medline and Google Scholar for systemic therapies related to Gorlin Syndrome (GS), expert opinion was specifically sought to provide detailed guidance on the use of SMOi in the management of GS.

Results:Intermittent dosing of SMOi and daily dosing had similar efficacy. Intermittent dosing resulted in better compliance with therapy. However, lack of tolerability limited long-term use of SMOi.

Limitations:There are few studies solely on the use of SMOi in GS. Included studies often had a subgroup of GS patients.

Conclusion:While the adverse events of SMOi, such as vismodegib and sonidegib, may result in their discontinuation during treatment of GS, intermittent dosing may improve compliance. Further research is needed to identify dosing strategies that minimize tumor burden while mitigating side effects to achieve maximum compliance.

Type

Article

PubMed ID

38950707

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