This protocol addresses patients with spinal cord hemangioblastoma arising in the context of von Hippel-Lindau (VHL) disease whose tumors are radiologically progressive and are currently asymptomatic or not amenable to surgical resection.
This approach does not apply to hemangioblastomas that are candidates for surgical removal.
Current evidence supports the use of a systemic small-molecule targeted agent for patients who meet this clinical profile. No size or multiplicity threshold is specified in the guideline.
The primary objective is objective radiologic response of the CNS or spinal cord hemangioblastoma — specifically, measurable reduction in tumor size.
DOI: 10.1093/noajnl/vdad153
Currently, belzutifan should be utilized in patients with progressive VHL-associated hemangioblastomas that are currently asymptomatic or unresectable.
However, it is not indicated for the treatment of hemangioblastomas that require surgical resection.
Recently, belzutifan has been used to successfully treat VHL-associated tumors.
Belzutifan is a small-molecule inhibitor of the HIF 2α subunit.
There is no direct guidance on the size or multiplicity of tumor thresholds for the use of the drug.
Specifically, it was shown to provide a 30% objective response rate in CNS hemangioblastomas over 21.8 months of median follow-up in 50 patients.
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