Regression of retinal capillary hemangioblastoma with systemic belzutifan in von Hippel–Lindau disease: a case report - Report - MDSpire

Regression of retinal capillary hemangioblastoma with systemic belzutifan in von Hippel–Lindau disease: a case report

  • By

  • Angelica Aja Dangca Alcoreza

  • Cheryn Song

  • Junyeop Lee

  • July 9, 2026

  • 0 min

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Clinical Report: Systemic Belzutifan Induces Regression of Retinal Capillary Hemangioblastoma

Overview

This case study reports the regression of retinal capillary hemangioblastoma in a patient with von Hippel–Lindau disease following treatment with systemic belzutifan. Notable reductions in lesion size, perfusion, and vascularity were observed four months after treatment initiation.

Background

Von Hippel–Lindau disease is a hereditary tumor syndrome that predisposes individuals to various tumors, including retinal capillary hemangioblastomas. These benign tumors are commonly associated with VHL and can lead to vision impairment. Effective treatment options are crucial for managing these lesions, particularly in patients with progressive disease.

Data Highlights

No numerical data or trial data presented in the article.

Key Findings

  • A 49-year-old female with VHL disease experienced regression of retinal capillary hemangioblastoma after systemic belzutifan treatment.
  • Four months post-treatment, a reduction in the size of retinal lesions was noted.
  • Decreased perfusion and vascularity of the lesions were observed following belzutifan therapy.
  • Belzutifan is a HIF-2α inhibitor approved for VHL-related tumors not requiring urgent surgery.
  • Previous treatment options for retinal capillary hemangioblastoma include laser therapy and cryotherapy.

Clinical Implications

Clinicians should consider the findings regarding systemic belzutifan for managing retinal lesions in patients with VHL.

Conclusion

Systemic belzutifan therapy may induce regression of retinal capillary hemangioblastomas in patients with von Hippel–Lindau disease.

Related Resources & Content

  1. Author(s)/Org, Source, Year -- Title
  2. The ASCO Post, Updated Findings With Belzutifan in von Hippel-Lindau Disease–Associated CNS Hemangioblastomas, 2024 -- Title
  3. Consensus Guidelines for Ocular Surveillance of von Hippel-Lindau Disease - PubMed, 2023 -- Title
  4. FDA Label, 2025 -- Title
  5. The ASCO Post — Updated Findings With Belzutifan in von Hippel-Lindau Disease–Associated CNS Hemangioblastomas
  6. Journal of Neuro-Oncology — Hemangioblastomatosis in a patient with von Hippel-Lindau disease
  7. The effect of oral belzutifan on retinal hemangioblastomas in von Hippel-Lindau syndrome - PubMed
  8. Consensus Guidelines for Ocular Surveillance of von Hippel-Lindau Disease - PubMed
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