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Belzutifan for Ocular Tumors in Patients with von Hippel-Lindau Disease
Farzad Jamshidi1, Samuel Tadros2, Lola Lozano3
1Department of Ophthalmology, University of Pittsburgh Medical Center, Pittsburgh, PA, USA.
Background:
Belzutifan is an orally administered small molecule inhibitor of HIF-2-alpha that has been approved for use in von Hippel-Lindau (VHL)-associated renal cell carcinoma, central nervous system (CNS) hemangioblastomas, and pancreatic neuro-endocrine tumors. While use of the drug for treatment of VHL-associated retinal hemangioblastomas (RH) remains off-label, numerous case reports, case series, and a clinical trial sub-analysis have demonstrated excellent results in using the drug to control these tumors.
Summary:
We review the literature that has been published on the use of belzutifan for RH in patients with VHL. These studies have described good efficacy for treating RH and preventing the development of new ocular tumors. The efficacy for juxtapapillary and macular tumors that can be difficult to treat has been particularly promising. Dose reductions are commonly required due to side effects which most commonly include anemia and fatigue.
Key Messages:
While early reports are encouraging, the optimal dose of the drug for controlling RH along with the duration of therapy, role as a neoadjuvant, and ways to incorporate use of the drug into the treatment and screening paradigms for VHL-associated ocular disease are evolving.
Insights
Belzutifan shows promise in treating von Hippel-Lindau (VHL)-associated retinal hemangioblastomas (RH). This HIF-2-alpha inhibitor effectively controls existing tumors and prevents new ones, though dose adjustments may be needed for side effects.
Area of Science:
- Ophthalmology
- Oncology
- Genetics
Background:
- Belzutifan, a HIF-2-alpha inhibitor, is approved for VHL-associated cancers.
- Its use for VHL-associated retinal hemangioblastomas (RH) is off-label but shows significant promise.
- Existing literature includes case reports, series, and trial sub-analyses demonstrating positive outcomes.
Purpose of the Study:
- To review and synthesize published literature on belzutifan's efficacy in treating RH in patients with VHL.
- To assess the drug's effectiveness in controlling existing RH and preventing new ocular tumor development.
- To highlight promising results for difficult-to-treat juxtapapillary and macular tumors.
Main Methods:
- Literature review of studies on belzutifan for RH in VHL patients.
- Analysis of reported efficacy in controlling tumors and preventing new growths.
- Examination of treatment outcomes, particularly for specific tumor locations.
Main Results:
- Belzutifan demonstrates good efficacy in treating RH and preventing new ocular tumors.
- The drug has shown particularly promising results for juxtapapillary and macular RH.
- Common side effects include anemia and fatigue, often necessitating dose reductions.
Conclusions:
- Belzutifan is an effective treatment option for VHL-associated RH.
- Optimal dosing, treatment duration, and integration into VHL screening paradigms require further investigation.
- Ongoing research is crucial to refine belzutifan's role in managing VHL-associated ocular disease.
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