American Association of Plastic Surgeons
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Use of Hedgehog Inhibitors in the Treatment of Gorlin Syndrome
Darren LePere, MD1, Alexis Lo, MD1, Rogerio Neves, MD, PHD, FACS, FSSO2.
1Penn State Hershey Medical Center, Hershey, PA, USA, 2Moffitt Cancer Center and Research Institute, Tampa, FL, USA.

PURPOSE: Gorlin Syndrome, or Nevoid Basal Cell Carcinoma Syndrome (NBCCS), is a rare genetic disorder characterized by multiple basal cell carcinomas (BCC). Although excisions have traditionally been used to decrease tumor burden, recent advancements in our understanding of Sonic Hedgehog (SHH) pathways and their mutations in NBCCS have shed light on treatments using immunomodulators. The purpose of this study was to explore the effects of SHH pathway inhibitors and their ability to reduce incidence of new BCCs and need for debulking surgeries.
METHODS: We performed a retrospective review of patients with NBCCS at our institution from 1999-2019. Each patient acted as their own control to compare incidence of new BCCs and surgeries conducted before and after initiation of treatment with a SHH pathway inhibitor.
RESULTS: Twenty-two patients met study criteria. Eighteen received treatment with vismodegib (81.8%), two with sonidegib (9.1%), and two with SUBA-itraconazole (9.1%). Prior to therapy, patients developed an average of 7.9 new BCC/year, as compared to 1.9 new BCC/year during therapy (76.3% reduction, p=0.051). Patients underwent an average of 2.3 surgeries/year before therapy and 0.6 surgeries/year with therapy (73.9% reduction, p=0.001).
CONCLUSION: Treatment with SHH pathway inhibitors resulted in a statistically significant reduction in the average number of surgeries for BCC/year, with a decrease in new BCC/year that trended toward significance (p=0.051) in our small study population. This reduction in annual BCCs and surgeries is amplified over a lifetime by lessening the severe physical and psychological toll that multiple resections and tumor burden have on these patients.


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