Pazopanib therapy for cerebellar hemangioblastomas in von Hippel-Lindau disease

Betty Y.S. Kim, Eric Jonasch, Ian E. McCutcheon

Research output: Contribution to journalArticlepeer-review

24 Scopus citations


Von Hippel-Lindau (VHL) disease is a genetically acquired multisystem tumor syndrome of the viscera and central nervous system (CNS). The most common tumors associated with this disease are histologically benign, slow-growing CNS hemangioblastomas affecting the retina, cerebellum, brainstem, spinal cord ornerve roots. With mean age at diagnosis of 30 years, CNS hemangioblastomas are usually the first manifestation of the disease. Ongoing clinical and radiological surveillance is required, with symptomatic lesions necessitating treatment. As tumor growth is inevitable during the lifetime of most VHL patients, and the multiplicity of tumors may preclude surgical cure, the search for effective therapies is ongoing. Here we provide the first report demonstrating clinical and radiological anti-tumor response using pazopanib, a small molecule multi-receptor tyrosine kinase inhibitor, in a patient with treatment-refractory VHL-associated CNS heman-gioblastoma. Treatment initiation with daily oral pazopanib (800 mg/day) resulted in significant neurologic improvement and radiologic tumor volume reduction.

Original languageEnglish (US)
Pages (from-to)145-149
Number of pages5
JournalTargeted Oncology
Issue number2
StatePublished - Jun 2012


  • Hemangioblastoma
  • Pazopanib
  • Von Hippel-Lindau disease

ASJC Scopus subject areas

  • Oncology
  • Cancer Research
  • Pharmacology (medical)


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