Response of a von Hippel-Lindau-associated optic nerve hemangioblastoma to belzutifan.

To demonstrate the response of a juxtapapillary retinal hemangioblastoma (JRH) to belzutifan in a patient with Von-Hippel-Lindau syndrome (VHL). This case report includes fundus photography and optical coherence tomography to compare a juxtapapillary retinal hemangioblastoma (JRH) before and 24 months after treatment with belzutifan. An 18-year-old woman with VHL was diagnosed with a juxtapapillary retinal hemangioblastoma (JRH) at age 13. The retinal lesion progressively worsened over 5 years and required two sessions of laser therapy with minimal improvement but loss of visual field. She was treated with the oral HIF2a-inhibitor, belzutifan, with minimal side effects. At 24 months of therapy her JRH has decreased in height from 1258 microns to 874 microns and width from 4448 µm to 3786 µm and vision is 20/20. Belzutifan demonstrates impressive clinical activity in VHL-associated JRH.