Clinical and molecular analysis of smoothened inhibitors in Sonic Hedgehog medulloblastoma.
Sonic Hedgehog
growth plate fusion
medulloblastoma
smoothened inhibitor
Journal
Neuro-oncology advances
ISSN: 2632-2498
Titre abrégé: Neurooncol Adv
Pays: England
ID NLM: 101755003
Informations de publication
Date de publication:
Historique:
entrez:
19
8
2021
pubmed:
20
8
2021
medline:
20
8
2021
Statut:
epublish
Résumé
Smoothened inhibitors (SMOi) have shown activity in Sonic Hedgehog (SHH) medulloblastoma, however this therapeutic class was not developed in children due to severe effects reported on growth. We hereby report long-term follow-up of young patients treated with SMOi for recurrent medulloblastoma. Clinical data on response and toxicity from patients treated with vismodegib or sonidegib from 2011 to 2019 for a SHH medulloblastoma were retrospectively reviewed. Methylation analysis and whole exome sequencing were performed whenever possible. All patients with a somatic Targeting SMO in mutated
Sections du résumé
BACKGROUND
BACKGROUND
Smoothened inhibitors (SMOi) have shown activity in Sonic Hedgehog (SHH) medulloblastoma, however this therapeutic class was not developed in children due to severe effects reported on growth. We hereby report long-term follow-up of young patients treated with SMOi for recurrent medulloblastoma.
METHODS
METHODS
Clinical data on response and toxicity from patients treated with vismodegib or sonidegib from 2011 to 2019 for a SHH medulloblastoma were retrospectively reviewed. Methylation analysis and whole exome sequencing were performed whenever possible.
RESULTS
RESULTS
All patients with a somatic
CONCLUSIONS
CONCLUSIONS
Targeting SMO in mutated
Identifiants
pubmed: 34409296
doi: 10.1093/noajnl/vdab097
pii: vdab097
pmc: PMC8367281
doi:
Types de publication
Journal Article
Langues
eng
Pagination
vdab097Informations de copyright
© The Author(s) 2021. Published by Oxford University Press, the Society for Neuro-Oncology and the European Association of Neuro-Oncology.
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