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Belzutifan was associated with substantial regression of von Hippel-Lindau-associated retinal capillary hemangioblastomas and absence of new lesions during on-treatment follow-up. These findings support a role for belzutifan in the management of juxtapapillary tumors and as adjunctive therapy for large or multifocal lesions. Frequent reactivation after treatment cessation suggests that continued administration may be required to maintain disease control.
Patients with JS ocular phenotypes present with oculomotor apraxia (80% of patients), strabismus (74%), nystagmus (72%), ptosis (44%), retinal degeneration (38%), chorioretinal coloboma (30%), optic nerve atrophy (22%), and abnormal retinal pigmentation (4.5%).
Short-term associations between bisphosphonate use and ocular inflammatory events varied by administration route and outcome. Increased uveitis risk was concentrated among intravenous formulations, particularly zoledronic acid, whereas no clear associations were found for scleritis or cystoid macular edema. These findings may inform monitoring for ocular symptoms among patients receiving intravenous bisphosphonates.
