title: Consensus recommendations for current treatments and accelerating clinical trials for patients with neurofibromatosis type 2.
abstract: Neurofibromatosis type 2 (NF2) is a tumor suppressor syndrome characterized by bilateral vestibular schwannomas (VS) which often result in deafness despite aggressive management. Meningiomas, ependymomas, and other cranial nerve and peripheral schwannomas are also commonly found in NF2 and collectively lead to major neurologic morbidity and mortality. Traditionally, the overall survival rate in patients with NF2 is estimated to be 38% at 20 years from diagnosis. Hence, there is a desperate need for new, effective therapies. Recent progress in understanding the molecular basis of NF2 related tumors has aided in the identification of potential therapeutic targets and emerging clinical therapies. In June 2010, representatives of the international NF2 research and clinical community convened under the leadership of Drs. D. Gareth Evans (University of Manchester) and Marco Giovannini (House Research Institute) to review the state of NF2 treatment and clinical trials. This manuscript summarizes the expert opinions about current treatments for NF2 associated tumors and recommendations for advancing therapies emerging from that meeting. The development of effective therapies for NF2 associated tumors has the potential for significant clinical advancement not only for patients with NF2 but for thousands of neuro-oncology patients afflicted with these tumors.
authors.affiliation: Johns Hopkins University, Baltimore, Maryland 21204, USA. jblakel3@jhmi.edu
authors.forename: Jaishri O
authors.initials: JO
authors.lastname: Blakeley
authors.forename: D Gareth
authors.initials: DG
authors.lastname: Evans
authors.forename: John
authors.initials: J
authors.lastname: Adler
authors.forename: Derald
authors.initials: D
authors.lastname: Brackmann
authors.forename: Ruihong
authors.initials: R
authors.lastname: Chen
authors.forename: Rosalie E
authors.initials: RE
authors.lastname: Ferner
authors.forename: C Oliver
authors.initials: CO
authors.lastname: Hanemann
authors.forename: Gordon
authors.initials: G
authors.lastname: Harris
authors.forename: Susan M
authors.initials: SM
authors.lastname: Huson
authors.forename: Abraham
authors.initials: A
authors.lastname: Jacob
authors.forename: Michel
authors.initials: M
authors.lastname: Kalamarides
authors.forename: Matthias A
authors.initials: MA
authors.lastname: Karajannis
authors.forename: Bruce R
authors.initials: BR
authors.lastname: Korf
authors.forename: Victor-Felix
authors.initials: VF
authors.lastname: Mautner
authors.forename: Andrea I
authors.initials: AI
authors.lastname: McClatchey
authors.forename: Harry
authors.initials: H
authors.lastname: Miao
authors.forename: Scott R
authors.initials: SR
authors.lastname: Plotkin
authors.forename: William
authors.initials: W
authors.lastname: Slattery
authors.forename: Anat O
authors.initials: AO
authors.lastname: Stemmer-Rachamimov
authors.forename: D Bradley
authors.initials: DB
authors.lastname: Welling
authors.forename: Patrick Y
authors.initials: PY
authors.lastname: Wen
authors.forename: Brigitte
authors.initials: B
authors.lastname: Widemann
authors.forename: Kim
authors.initials: K
authors.lastname: Hunter-Schaedle
authors.forename: Marco
authors.initials: M
authors.lastname: Giovannini
mesh_terms: D002986:Clinical Trials as Topic; D023381:Endpoint Determination; D006801:Humans; D008579:Meningioma / Q000175:diagnosis / Q000628:therapy; D016518:Neurofibromatosis 2 / Q000175:diagnosis* / Q000235:genetics / Q000628:therapy*; D016634:Radiosurgery; D012107:Research Design; D059039:Standard of Care