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Emily Duan
Emily Duan

Public Documents 1
Pediatric Patients with Von Hippel-Lindau and Hemangioblastomas Treated Successfully...
Emily Duan
Michael Robinson

Emily Duan

and 9 more

June 12, 2024
Hemangioblastomas are the most common tumor associated with Von Hippel-Lindau (VHL) and a leading cause of mortality. We present five pediatric patients with VHL-associated hemangioblastomas treated with belzutifan, a HIF2a inhibitor. Three patients were started on belzutifan due to vision loss from progressive retinal hemangioblastomas. Within 1 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 patients with VHL-associated hemangioblastomas.

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