Pediatric Patients with Von Hippel-Lindau and Hemangioblastomas Treated
Successfully with Belzutifan
Abstract
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.