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Updated: Feb 25, 2026

Cell Population Analyses During Skin Carcinogenesis
Published on: August 21, 2013
Risk of cutaneous squamous cell carcinoma after treatment of basal cell carcinoma with vismodegib
Tina Bhutani1, Michael Abrouk1, Camelia S Sima2
1Department of Dermatology, University of California San Francisco, San Francisco, California.
Background:
Vismodegib is a first-in-class agent targeting the hedgehog signaling pathway for treatment of patients with locally advanced basal cell carcinoma (BCC) and metastatic BCC. There have been concerns about the development of squamous cell carcinoma (SCC) in patients treated with this drug.
Objective:
We sought to determine whether treatment with vismodegib is associated with an increase in the risk of cutaneous SCC.
Methods:
In this retrospective cohort study, patients treated with vismodegib as part of phase I and II clinical studies were compared with participants from the University of California, San Francisco, Nonmelanoma Skin Cancer Cohort who received standard therapy for primary BCC. In total, 1675 patients were included in the analysis, and the development of SCC after vismodegib exposure was assessed.
Results:
The use of vismodegib was not associated with an increased risk of subsequent development of SCC (adjusted hazard ratio, 0.57; 95% confidence interval, 0.28-1.16). Covariates including age, sex, history of previous nonmelanoma skin cancer, and number of visits per year were significantly associated with the development of SCC.
Limitations:
A limitation of the study was that a historic control cohort was used as a comparator.
Conclusions:
Vismodegib was not associated with an increased risk of subsequent SCC when compared with standard surgical treatment of BCC.
Insights
Vismodegib, a hedgehog pathway inhibitor for basal cell carcinoma (BCC), did not increase the risk of squamous cell carcinoma (SCC) development. This finding is based on a study comparing vismodegib-treated patients with those receiving standard BCC therapy.
Area of Science:
- Oncology
- Dermatology
- Pharmacology
Background:
- Vismodegib is a targeted therapy for advanced basal cell carcinoma (BCC) that inhibits the hedgehog signaling pathway.
- Concerns exist regarding a potential increased risk of secondary cutaneous squamous cell carcinoma (SCC) in patients treated with vismodegib.
Purpose of the Study:
- To investigate the association between vismodegib treatment and the risk of developing cutaneous SCC.
- To evaluate the safety profile of vismodegib concerning SCC development in BCC patients.
Main Methods:
- Retrospective cohort study design comparing patients treated with vismodegib in clinical trials.
- Comparison group included patients receiving standard therapy for primary BCC from a nonmelanoma skin cancer cohort.
- Analysis included 1675 patients assessing SCC development post-vismodegib exposure.
Main Results:
- Vismodegib treatment was not associated with an increased risk of subsequent SCC (adjusted hazard ratio, 0.57; 95% CI, 0.28-1.16).
- Factors such as age, sex, prior nonmelanoma skin cancer history, and visit frequency were significant predictors of SCC development.
- No statistically significant increase in SCC risk was observed with vismodegib compared to standard treatments.
Conclusions:
- Vismodegib is not linked to a higher risk of developing SCC in patients with BCC.
- The study utilized a historical control cohort, which is a noted limitation.
- Results suggest vismodegib can be safely used for BCC treatment without elevating SCC risk compared to standard care.
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