Mechanisms, indications and results of salvage systemic therapy for sporadic and von Hippel-Lindau related hemangioblastomas of the central nervous system

Crit Rev Oncol Hematol. 2013 Apr;86(1):69-84. doi: 10.1016/j.critrevonc.2012.10.001. Epub 2012 Nov 11.


Hemangioblastomas (HBs) are rare indolent vascular tumors that may occur sporadically or in association with von Hippel-Lindau (VHL) disease. Total neurosurgical resection is the standard upfront approach providing long-term tumor control. At time of tumor recurrence, second surgery, radiosurgery or radiotherapy are the main therapeutic strategies. Limited information is available on the role of pharmacological strategies. Anti-angiogenic agents, particularly multitarget tyrosine kinase inhibitors (semaxanib, sunitinib, vatalanib), thalidomide and interferon alfa-2a are currently the most widely studied strategies to prolonge disease stability. Salvage therapy with anti-angiogenetic drugs may be of benefit in some patients who are not suitable for surgery, radiosurgery or radiotherapy, with progressive or recurrent hemangioblastoma especially those located in retina, since anti-angiogenetic therapy may delay tumor progression. This strategy warrants prospective evaluation in a clinical trial.

Publication types

  • Review

MeSH terms

  • Antineoplastic Agents / therapeutic use*
  • Central Nervous System Neoplasms / diagnosis
  • Central Nervous System Neoplasms / drug therapy
  • Central Nervous System Neoplasms / etiology
  • Central Nervous System Neoplasms / genetics
  • Cerebellar Neoplasms / diagnosis
  • Cerebellar Neoplasms / drug therapy*
  • Cerebellar Neoplasms / etiology
  • Cerebellar Neoplasms / genetics
  • Hemangioblastoma / diagnosis
  • Hemangioblastoma / drug therapy*
  • Hemangioblastoma / etiology
  • Hemangioblastoma / genetics
  • Humans
  • Salvage Therapy*
  • Signal Transduction / genetics
  • Signal Transduction / physiology
  • Treatment Outcome
  • von Hippel-Lindau Disease / complications
  • von Hippel-Lindau Disease / diagnosis
  • von Hippel-Lindau Disease / drug therapy*
  • von Hippel-Lindau Disease / genetics


  • Antineoplastic Agents