Related Experiment Video
Updated: Jun 22, 2025
Author Spotlight: Oral Candida Diagnosis to Advance Clinical Treatment Regimen for pSS Patients
Published on: March 1, 2024
Oral smoothened inhibitors for Gorlin syndrome: A clinical review
Alexandra Baczynski1, Brian Cahn2, Brandon Worley3
1Department of Dermatology, Rush University Medical Center, Chicago, Illinois.
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.
Insights
Smoothened inhibitors (SMOi) show promise for Gorlin syndrome (GS) treatment. Intermittent dosing may improve patient compliance while maintaining similar efficacy to daily regimens, despite potential adverse events.
Area of Science:
- Oncology
- Dermatology
- Genetics
Background:
- Basal cell carcinoma is a common skin cancer.
- Gorlin syndrome (GS) is a rare genetic disorder predisposing individuals to multiple basal cell carcinomas.
- Smoothened inhibitors (SMOi) are effective for basal cell carcinoma but lack specific guidelines for GS.
Purpose of the Study:
- To review the clinical efficacy of SMOi in Gorlin syndrome.
- To offer practical guidance for clinicians treating GS patients with SMOi.
- To identify future research directions for SMOi in GS.
Main Methods:
- Comprehensive literature search of existing publications.
- Inclusion of expert opinion to supplement findings.
- Review focused on clinical response and adverse events of SMOi in GS.
Main Results:
- Intermittent dosing of SMOi demonstrates comparable efficacy to daily dosing in GS.
- Adverse events associated with SMOi can lead to treatment discontinuation.
- Intermittent dosing may enhance treatment compliance in GS patients.
Conclusions:
- SMOi are a viable treatment option for Gorlin syndrome.
- Intermittent SMOi dosing is a potentially effective strategy for improving compliance.
- Further research is needed to optimize SMOi therapy for GS.
Related Concept Videos
02:23Abnormal Proliferation
01:20The Retinoblastoma Gene
The first-ever tumor suppressor gene called Rb was identified in retinoblastoma - a rare eye tumor in children. In inherited forms of the disease, a child inherits one defective copy of the Rb gene, which predisposes them to retinoblastoma. However,...