Efficacy of Oral Belzutifan for Retinal Capillary Hemangioblastomas in von Hippel-Lindau Disease
In brief
Belzutifan shrank retinal tumor area 75% at one year in von Hippel-Lindau disease
In this 33-patient case series, average active retinal tumor area per eye fell 75% after one year of belzutifan, and no new tumors appeared during treatment. More than 70% of eyes showed signs of relapse after patients stopped therapy, suggesting control may depend on continued treatment; larger studies are needed to confirm these findings.
- Journal
- Ophthalmology. Retina (Q1)
- Published
- 23 October 2026
- Study design
- Case series / case report
- Evidence level
- Level 4, Very Low (CEBM 4)
- Authors
- Marco H Ji, Alisa T Thavikulwat, Elvira Agrón, Mélanie Hébert, Asmita Indurkar, Cameron Ward, et al.
- PMID
- 42918417
- DOI
- 10.1016/j.oret.2026.10.008
Why clinicians should know about it
- Picked for Ophthalmology (paper of the day, 26 October 2026): Clinical belzutifan outcomes for retinal capillary hemangioblastomas
Abstract
OBJECTIVE: To evaluate the efficacy of oral belzutifan on retinal capillary hemangioblastomas (RCHs) in patients with von Hippel-Lindau (VHL) disease. DESIGN: Single-center, retrospective case series. PARTICIPANTS: Thirty-three patients (42 eyes) with VHL disease and at least one measurable RCH who were treated with oral belzutifan (40-120 mg/day) and examined at the National Eye Institute with at least one follow-up visit. MAIN OUTCOMES: Change in total active lesion area, juxtalesional exudation, and feeder and draining vessel tortuosity and dilation. RESULTS: A total of 85 active lesions (42 eyes) were identified at baseline prior to starting belzutifan and were followed for an average of 21.8 ± 14.7 months while on belzutifan. Mean ± standard deviation total active lesion area per eye decreased significantly from 5.8 ± 9.7 mm2 at baseline to 1.2 ± 2.3 mm2 at 1 year (P <0.001) and to 0.5 ± 0.8 mm2 (P <0.001) at 2 years. This change represented an average decrease of 75.0 ± 32.4% at 1 year and 79.5 ± 29.8% at 2 years. No new RCHs were observed during active treatment. However, among patients who discontinued therapy, over 70% of eyes exhibited signs of relapse, including increased lesion area. CONCLUSIONS: Belzutifan was associated with substantial regression of VHL-associated RCHs 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.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.