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  • Updated 09.02.2022
  • Released 02.17.1999
  • Expires For CME 09.02.2025




Hemangioblastoma is a relatively uncommon tumor that most often arises in the cerebellum, brainstem, spinal cord, or cauda equina. Hemangioblastomas occur sporadically as a single lesion and also occur as multiple tumors in persons with von Hippel-Lindau (VHL) disease, an inheritable genetic disorder. The mainstay of treatment for hemangioblastoma continues to be surgical resection. Stereotactic radiosurgery is used in selective situations. The HIF2α inhibitor, belzutifan (Welireg), has received FDA approval for von Hippel-Lindau-associated hemangioblastoma. In this article, the author reviews the clinical features, molecular genetics, and current therapies for hemangioblastoma.

Key points

• Hemangioblastomas most often arise in the cerebellum, brainstem, spinal cord, and cauda equina.

• One third to one half of patients with hemangioblastoma have von Hippel-Lindau (VHL) disease whereas at least two thirds of persons with von Hippel-Lindau disease develop 1 or more hemangioblastomas over their lifetime.

• Most patients presenting with a hemangioblastoma should be evaluated for von Hippel-Lindau disease.

• Treatment options for sporadically occurring hemangioblastoma or for symptomatic hemangioblastoma in von Hippel-Lindau disease patients include surgical resection or radiation therapy (usually stereotactic radiosurgery) in selected patients.

• The HIF2α inhibitor, belzutifan, has been approved for the treatment of von Hippel-Lindau-associated hemangioblastomas.

Historical note and terminology

Hughlings Jackson described the index case of cerebellar hemangioblastoma in 1872 (36). In 1904, Eugen von Hippel described the histology of retinal hemangioblastoma (also known as retinal angioma) and recognized its familial occurrence (82). Arvid Lindau in 1926 reported the combination of hemangioblastomas of the retina and central nervous system with other systemic lesions as a unified familial disease process (52). The classic paper by Melmon and Rosen in 1964 reviewed a large kindred with the disorder they codified as "von Hippel-Lindau disease" (58). The genetic linkage of von Hippel-Lindau disease to chromosome 3p was established in the 1970s and 1980s. The von Hippel-Lindau disease gene was identified and cloned in 1993 (48).

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