Available Therapies for Patients with Neurofibromatosis-Related Nervous System Tumors
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Neuro-oncology (GJ Lesser, Section Editor)
Available Therapies for Patients with Neurofibromatosis-Related Nervous System Tumors Roy E Strowd III, MD, MEd, MS Address 1 Department of Neurology, Internal Medicine, Section on Hematology and Oncology, and the Translational Sciences Institute, Wake Forest School of Medicine, 1 Medical Center Boulevard, Winston Salem, NC, 27157, USA Multidisciplinary Neurofibromatosis Clinic, Wake Forest Baptist Comprehensive Cancer Center, Wake Forest School of Medicine, 1 Medical Center Boulevard, Winston Salem, NC, 27157, USA Email: [email protected]
* Springer Science+Business Media, LLC, part of Springer Nature 2020
Patients with Neurofibromatosis-Related Nervous System Tumors This article is part of the Topical Collection on Neuro-oncology Keywords Neurofibromatosis type 1 I Neurofibromatosis type 2 I Schwannomatosis I Neurocutaneous disorders I Phakomatoses
Opinion statement Though the majority of nervous system tumors are sporadic, several clinically relevant genetic syndromes are associated with a predisposition to tumors of the central and peripheral nervous system including neurofibromatosis type 1 (NF1), type 2 (NF2), and schwannomatosis (SWN). These represent prototypical tumor suppressor syndromes where loss of a tumor suppressor gene-protein impairs the cell’s ability to regulate cell proliferation. While clinical manifestations vary widely for each of these syndromes, tumors arising in the peripheral nerve sheath are a unifying feature. Clinical clues should prompt the clinician to recognize the underlying genetic syndrome and screen for associated tumors including, among others, plexiform neurofibromas and gliomas in NF1 and vestibular schwannomas, meningiomas, and spinal ependymomas in NF2. Improvements in mechanistic understanding of how the genetic mutations that underlie these syndromes contribute to tumor formation have led to new advances in targeted therapies. MEK inhibitors have shown promise for treating progressive plexiform neurofibromas in NF1. Bevacizumab has been shown to improve hearing and treat vestibular schwannomas in NF2. This article reviews the currently available data on management of tumors associated with these three syndromes.
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Curr. Treat. Options in Oncol.
(2020) 21:81
Introduction Neurofibromatosis type 1 (NF1), type 2 (NF2), and schwannomatosis (SWN) are rare tumor suppressor syndromes which predispose patients to tumors of the brain, spinal cord, or peripheral nerves. NF1 is the most common of these conditions with an estimated incidence of 1:3000 [1]. Comparatively, NF2 and SWN are rare with incidences of 1:28,000 and 1:100,000, respectively [2]. These are genetic disorders which follow an autosomal dominant inheritance pattern. The natural history can be highly variable from patient to patient and include both nervous and extra-nervous system manifestations. While tumors can develop throughout the central and peripheral nervous systems, peripheral nerve sheath tumors (PNSTs) are a unifying feature of each co
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