Neurofibromatosis type 1-associated gliomas and other tumors: A new pathway forward?

• Published in: Pediatric hematology oncology journal Vol. 8; no. 2; pp. 129 - 135
• Main Authors: Webster Carrion, Andrea, Shah, Amish C., Kotch, Chelsea
• Format: Journal Article
• Language: English
• Published: Elsevier B.V 01-06-2023; Elsevier
• Subjects: Low-grade glioma; Nerve sheath tumor; Neurofibromatosis; Optic pathway glioma; Optic pathway glioma; MRI; Neurofibromatosis; Low-grade glioma; OPG; NF1; Nerve sheath tumor; CNS; LGG; PN; MPNST
• ISSN: 2468-1245; 2468-1245
• DOI: 10.1016/j.phoj.2023.05.002

Neurofibromatosis type 1 (NF1) is an autosomal dominant cancer predisposition syndrome caused by germline alterations of the NF1 gene. Patients with NF1 are at increased risk for developing benign and malignant tumors, such as optic pathway glioma and peripheral nerve sheath neoplasms. The number of effective therapies for neurofibromatosis-related tumors has been historically limited. However, there have been recent advances in understanding and developing targeted therapies for these tumors, such as mitogen-activated kinase inhibitors and multiple tyrosine kinase inhibitors. Herein, we review the most prevalent tumors occurring in patients with NF1, updated management guidelines, and explore the approach to therapy in resource-limited settings.