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Master Thesis

Le vismodegib : revue de la littérature et expérience dans la prise en charge du cancer basocellulaire au CHU de Bordeaux

Abstract : Basal cell carcinoma (BCC) is the most common skin cancer. Death rate from BCC is low as these tumor metastasize extremely rarely. Surgery is the treatment of choice for BCC. Genetic mutations have been identified in pathogenesis of BCC such as alteration in Patched receptor or activating mutation of Smoothened protein. These mutations lead to overactivation of Hedgehog (Hh) signalling pathway, which plays a critical role during embryogenesis and tissue homeostasis in adults. Vismodegib is the first-in-class Hh pathway inhibitor approved by European Medicines Agency (EMA), after a single phase II clinical trial, and indicated for advanced and metastasis BCC management when surgery and radiation therapy are considered inappropriate. We conducted a retrospective observational study on patients treated by vismodegib in dermatologic unit of Bordeaux University Hospital. Our results are broadly consistent with literature data, they show a moderate efficacy associated with a poor tolerance. Moreover, these data suggest a potential interindividual variability of vismodegib response. Clinical, histologic or demographic factor associated with complete response has not been identified yet. Owing to a moderate efficacy and duration of treatment limited by adverse effect occurrences, clinicians proposed news therapeutic strategies for vismodegib: 1) a discontinued strategy 2) use of vismodegib as neoadjuvant to surgery and radiotherapy. News therapeutic strategies must be assess to determine the position of vismodegib in BCC therapeutic arsenal.
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Master Thesis
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Contributor : Bu Carreire Université de Bordeaux <>
Submitted on : Friday, February 13, 2015 - 9:40:34 AM
Last modification on : Wednesday, August 23, 2017 - 4:40:20 PM
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  • HAL Id : dumas-01116312, version 1



Caroline Streicher. Le vismodegib : revue de la littérature et expérience dans la prise en charge du cancer basocellulaire au CHU de Bordeaux. Sciences pharmaceutiques. 2014. ⟨dumas-01116312⟩



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