Belzutifan-induced tumor regression in sporadic hemangioblastoma: a case report and literature review - Takeaways - MDSpire

Belzutifan-induced tumor regression in sporadic hemangioblastoma: a case report and literature review

  • By

  • Rebekka E. Hooks

  • Niket Yadav

  • Mark Willy L. Mondia

  • Georgios Mantziaris

  • Alaa Saleh

  • Anna Vi Jones

  • Matthew McCord

  • Melike Mut

  • Ashok R. Asthagiri

  • Benjamin W. Purow

  • February 14, 2026

  • 0 min

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  • 1

    Hemangioblastomas are WHO grade 1 tumors, primarily found in the CNS, constituting 1-2% of intracranial tumors and up to 10% of spinal cord tumors.

  • 2

    Belzutifan is a selective HIF-2α inhibitor that disrupts oncogenic transcription, showing efficacy in VHL-associated hemangioblastomas and other neoplasms.

  • 3

    The objective response rates for CNS hemangioblastomas treated with belzutifan range from 44% to 76%, with a median disease control rate of 90%.

  • 4

    This article presents the first case of tumor regression in a sporadic CNS hemangioblastoma treated with belzutifan, suggesting HIF-2α pathway dependence.

  • 5

    Management of hemangioblastomas is challenging, with surgical resection being the primary treatment, while belzutifan offers a promising systemic therapy option.

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