Pediatric patients with von Hippel–Lindau and hemangioblastomas treated successfully with belzutifan

Abstract

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.