Single-center retrospective study of belzutifan for VHL-associated central nervous system hemangioblastoma

Belzutifan is a selective inhibitor of hypoxia-inducible factor 2α (HIF-2a) that can be used as targeted therapy for Von Hippel-Lindau (VHL) syndrome associated tumors. There is limited real-world evidence regarding safety and efficacy of belzutifan in CNS hemangioblastoma. A single-center retrospective trial at the Mayo Clinic (Rochester, MN) evaluated belzutifan in four adult patients with VHL and craniospinal CNS hemangioblastoma not amenable to surgical resection. The treatment was safe and well-tolerated and induced strong disease response in all patients.