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Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome)

Nevoid basal cell carcinoma syndrome (NBCCS, Gorlin syndrome) is a rare genetic condition characterized by the early development of numerous cutaneous basal cell carcinomas (BCCs). Although most BCCs are surgically treated with total resection, some of the lesions may proceed to a locally advanced o...

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Autores principales: Venturi, Federico, Trane, Luciana, Silvestri, Flavia, Zuccaro, Biancamaria, Magnaterra, Elisabetta, De Giorgi, Vincenzo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10549827/
https://www.ncbi.nlm.nih.gov/pubmed/37799353
http://dx.doi.org/10.1002/ski2.260
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author Venturi, Federico
Trane, Luciana
Silvestri, Flavia
Zuccaro, Biancamaria
Magnaterra, Elisabetta
De Giorgi, Vincenzo
author_facet Venturi, Federico
Trane, Luciana
Silvestri, Flavia
Zuccaro, Biancamaria
Magnaterra, Elisabetta
De Giorgi, Vincenzo
author_sort Venturi, Federico
collection PubMed
description Nevoid basal cell carcinoma syndrome (NBCCS, Gorlin syndrome) is a rare genetic condition characterized by the early development of numerous cutaneous basal cell carcinomas (BCCs). Although most BCCs are surgically treated with total resection, some of the lesions may proceed to a locally advanced or metastatic stage. Systemic treatment with a hedgehog inhibitors (HHIs) such as Vismodegib or Sonidegib is indicated in this population. We report cases of two patients with confirmed diagnoses of NBCCS. Both patients had undergone multiple surgical excisions and had been treated with oral Vismodegib 150 mg/day for a locally advanced tumour. They both discontinued the therapy due to its specific adverse effects (AEs) and are now being treated with oral Sonidegib, which has had better tolerability and a complete response. The aims of this report was to demonstrate the efficacy of HHI treatment with Sonidegib in patients with NCBBS who had previously treated with Vismodegib but discontinued it because of its specific AEs. Our experience in two patients shows that Sonidegib can be considered in Gorlin patients intolerant but responding to Vismodegib.
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spelling pubmed-105498272023-10-05 Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome) Venturi, Federico Trane, Luciana Silvestri, Flavia Zuccaro, Biancamaria Magnaterra, Elisabetta De Giorgi, Vincenzo Skin Health Dis Case Reports Nevoid basal cell carcinoma syndrome (NBCCS, Gorlin syndrome) is a rare genetic condition characterized by the early development of numerous cutaneous basal cell carcinomas (BCCs). Although most BCCs are surgically treated with total resection, some of the lesions may proceed to a locally advanced or metastatic stage. Systemic treatment with a hedgehog inhibitors (HHIs) such as Vismodegib or Sonidegib is indicated in this population. We report cases of two patients with confirmed diagnoses of NBCCS. Both patients had undergone multiple surgical excisions and had been treated with oral Vismodegib 150 mg/day for a locally advanced tumour. They both discontinued the therapy due to its specific adverse effects (AEs) and are now being treated with oral Sonidegib, which has had better tolerability and a complete response. The aims of this report was to demonstrate the efficacy of HHI treatment with Sonidegib in patients with NCBBS who had previously treated with Vismodegib but discontinued it because of its specific AEs. Our experience in two patients shows that Sonidegib can be considered in Gorlin patients intolerant but responding to Vismodegib. John Wiley and Sons Inc. 2023-06-14 /pmc/articles/PMC10549827/ /pubmed/37799353 http://dx.doi.org/10.1002/ski2.260 Text en © 2023 The Authors. Skin Health and Disease published by John Wiley & Sons Ltd on behalf of British Association of Dermatologists. https://creativecommons.org/licenses/by/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
spellingShingle Case Reports
Venturi, Federico
Trane, Luciana
Silvestri, Flavia
Zuccaro, Biancamaria
Magnaterra, Elisabetta
De Giorgi, Vincenzo
Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome)
title Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome)
title_full Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome)
title_fullStr Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome)
title_full_unstemmed Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome)
title_short Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome)
title_sort tolerance of sonidegib after intolerance of vismodegib—experience in two patients with nevoid basal cell carcinoma syndrome (gorlin syndrome)
topic Case Reports
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10549827/
https://www.ncbi.nlm.nih.gov/pubmed/37799353
http://dx.doi.org/10.1002/ski2.260
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