Pediatric patients with von Hippel-Lindau and hemangioblastomas treated successfully with belzutifan.
belzutifan
hemangioblastoma
pediatric
von Hippel–Lindau
Journal
Pediatric blood & cancer
ISSN: 1545-5017
Titre abrégé: Pediatr Blood Cancer
Pays: United States
ID NLM: 101186624
Informations de publication
Date de publication:
16 Oct 2024
16 Oct 2024
Historique:
revised:
24
08
2024
received:
29
04
2024
accepted:
23
09
2024
medline:
17
10
2024
pubmed:
17
10
2024
entrez:
17
10
2024
Statut:
aheadofprint
Résumé
Hemangioblastoma is the most common tumor associated with von Hippel-Lindau (VHL), and are a leading cause of mortality. We present five pediatric patients with VHL-associated hemangioblastomas treated with belzutifan, a hypoxia-inducible factor 2a (HIF2a) inhibitor. Three patients were started on belzutifan due to vision loss from progressive retinal hemangioblastomas. Within one year of treatment, all three patients had improvement in hemangioblastoma size and visual acuity. For patients with intracranial lesions, belzutifan resulted in an improvement in neurologic symptoms and hemangioblastoma size. Four patients experienced grade 1-2 anemia and two patients required a dose reduction. Our report suggests that belzutifan can be an effective therapy for pediatric, adolescent, and young adult patients with VHL-associated hemangioblastomas.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e31371Informations de copyright
© 2024 The Author(s). Pediatric Blood & Cancer published by Wiley Periodicals LLC.
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