Belzutifan as the Primary Treatment of Bilateral Juxtapapillary Retinal Hemangioblastoma in a Patient With Von Hippel-Lindau Disease.

Abstract

Purpose: To describe the efficacy of belzutifan as a treatment for juxtapapillary retinal hemangioblastomas in patients with von Hippel-Lindau disease. Methods: A case and its findings were analyzed, and a systematic literature review was conducted using PubMed and Ovid MEDLINE. Results: At a routine follow-up, a 63-year-old woman with a history of von Hippel-Lindau disease and slowly progressive bilateral juxtapapillary retinal hemangioblastomas presented with decreased visual acuity (VA) in the right eye resulting from significant lesion growth and an increase in central macular edema and exudate. Oral belzutifan therapy was initiated. A significant bilateral regression and decrease in tumor size, improved macular thickening and edema, and improved VA were seen over a 6-month period. Conclusions: The current literature on the therapeutic effects of oral belzutifan is limited; however, recent reports have been promising. This case shows the potential efficacy of belzutifan as a first-line treatment for vision-threatening juxtapapillary retinal hemangioblastomas in patients with von Hippel-Lindau disease.