Neurofibromatosis type 1 (NF1) is an autosomal dominant cancer predisposition syndrome characterized by the development of both central and peripheral nervous system tumors. Low-grade glioma (LGG) is the most prevalent central nervous system tumor occurring in children with NF1, arising most frequently within the optic pathway, followed by the brainstem. Historically, treatment of NF1-LGG has been limited to conventional cytotoxic chemotherapy and surgery. Despite treatment with chemotherapy, a subset of children with NF1-LGG fail initial therapy, have a continued decline in function, or recur. The recent development of several preclinical models has allowed for the identification of novel, molecularly targeted therapies. At present, exploration of these novel precision-based therapies is ongoing in the preclinical setting and through larger, collaborative clinical trials. Herein, we review the approach to surveillance and management of NF1-LGG in children and discuss upcoming novel therapies and treatment protocols.
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http://dx.doi.org/10.1007/s00381-024-06430-8 | DOI Listing |
Front Oncol
December 2024
Department of Pediatric Hematology-Oncology and Blood and Marrow Transplant, Cleveland Clinic, Cleveland, OH, United States.
Pediatric low-grade gliomas (pLGGs) are the most common brain tumors in children. Many patients with unresectable tumors experience recurrence or long-term sequelae from standard chemotherapeutics. This mini-review explores the emerging role of MEK inhibitors in the management of pLGGs, highlighting their potential to transform current treatment paradigms.
View Article and Find Full Text PDFSci Rep
January 2025
Nuclear Medicine Department, Medical University of Warsaw, Banacha 1a, 02-097, Warsaw, Poland.
PET/CT targeting prostate-specific membrane antigen (PSMA) is commonly used in patients with prostate cancer. PSMA has been found in other solid tumours, including primary brain tumours. The aim of this study was to evaluate the usefulness of [Ga]Ga-PSMA-11 PET/CT for preoperative diagnosis and 2-year prognosis.
View Article and Find Full Text PDFJ Neurooncol
January 2025
Department of Neurosurgery, NYU Langone Health and NYU Grossman School of Medicine, 530 1st Avenue, Skirball Suite 8R, New York, NY, 10016, USA.
Unlabelled: QUESTIONS AND RECOMMENDATIONS FROM THE PRIOR VERSION OF THESE GUIDELINES WITHOUT CHANGE: TARGET POPULATION: Adult patients (age ≥ 18 years) who have suspected low-grade diffuse glioma.
Question: What are the optimal neuropathological techniques to diagnose low-grade diffuse glioma in the adult?
Recommendation: Level I Histopathological analysis of a representative surgical sample of the lesion should be used to provide the diagnosis of low-grade diffuse glioma. Level III Both frozen section and cytopathologic/smear evaluation should be used to aid the intra-operative assessment of low-grade diffuse glioma diagnosis.
Nat Commun
January 2025
Center for Data-Driven Discovery in Biomedicine (D3b), The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Front Pediatr
December 2024
Department of Neurosurgery, Tsinghua University Yuquan Hospital (Tsinghua University Hospital of Integrated Traditional Chinese and Western Medicine), Beijing, China.
Purpose: This study aims to summarize the characteristics of children under three years old (≤3 years) with central nervous system (CNS) tumors and to investigate the factors that influence their overall survival (OS) time.
Methods: We treated 171 pediatric patients (≤3 years) with CNS tumors at Yuquan Hospital of Tsinghua University from January 2016 to June 2023. Of these, 162 cases were successfully followed up.
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