title: Mechanisms, indications and results of salvage systemic therapy for sporadic and von Hippel-Lindau related hemangioblastomas of the central nervous system.
abstract: 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.
authors.affiliation: Department of Neurosurgery and Radiosurgery, IRCCS San Raffaele Scientific Institute, Milan, Italy. jody.capitanio@gmail.com
authors.forename: Jody Filippo
authors.initials: JF
authors.lastname: Capitanio
authors.forename: Elena
authors.initials: E
authors.lastname: Mazza
authors.forename: Micaela
authors.initials: M
authors.lastname: Motta
authors.forename: Pietro
authors.initials: P
authors.lastname: Mortini
authors.forename: Michele
authors.initials: M
authors.lastname: Reni
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D016543:Central Nervous System Neoplasms / Q000175:diagnosis / Q000188:drug therapy / Q000209:etiology / Q000235:genetics; D002528:Cerebellar Neoplasms / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology / Q000235:genetics; D018325:Hemangioblastoma / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology / Q000235:genetics; D006801:Humans; D016879:Salvage Therapy; D015398:Signal Transduction / Q000235:genetics / Q000502:physiology; D016896:Treatment Outcome; D006623:von Hippel-Lindau Disease / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy* / Q000235:genetics
chemical_list: D000970:Antineoplastic Agents