Retinal hemangioblastoma may be partially reduced with belzutifan treatment

Ocular Pathology/Oncology, Retina/Vitreous

Belzutifan (Welireg) was used in one young patient to partially reduce hemangioblastoma size and vascularity.

Study design

This is a single interventional case report of a 24-year-old patient with Von-Hippel-Lindau syndrome and central nervous system hemangioblastomas treated with belzutifan for 6 months.


The authors show partial regression of retinal hemangioblastomas in the left eye of the 24-year-old patient after 6 months of treatment with belzutifan, which is a hypoxia-inducible factor-2⍺ inhibitor. The retinal tumors showed decreased size and vascularity with reduction in size and tortuosity of the feeding and draining vessels.


This is a single case report with a limited follow-up duration of 6 months. The findings need to be confirmed on a larger number of patients and with longer follow-up to determine the long-term efficacy and possible ocular and systemic side effects of the treatment.

Clinical significance

Current modalities for treatment of retinal hemangioblastoma include laser photocoagulation, cryotherapy, photodynamic therapy, intravitreal injections of anti-VEGF agents or steroid, and pars plana vitrectomy. Unfortunately, these treatment techniques are not always effective and can be associated with serious complications. Treatment of retinal hemangioblastoma, especially when large and multiple, can therefore be challenging and may result in poor visual outcome, or even loss of the eye in some cases. The availability of some form of systemically administered treatment that will cause partial or complete regression of these tumors, as seen in the current case report, is an important addition to the armamentarium for treatment of retinal capillary hemangioblastoma and can improve the treatment outcome of these visually threatening tumors.

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