Decoding optic pathway gliomas: Molecular insights and emerging therapies.

Optic Pathway Gliomas (OPGs) are low-grade astrocytic tumors primarily affecting children, constituting 2 % of cerebral gliomas, with 90 % diagnosed before age 20. These tumors occur sporadically or in association with neurofibromatosis type 1 (NF1), where they often show a better prognosis. OPGs are classified by location-along the anterior (optic nerve) or posterior (chiasm/hypothalamus) visual pathway-and NF1 association. Pathologically, they are linked to BRAF alterations and, in NF1, neurofibromin loss, driving variable growth from indolent to aggressive, particularly in adults. Symptoms like visual loss, proptosis, and endocrinopathies depend on tumor site, with MRI as the diagnostic cornerstone, supplemented by biopsy when needed. The clinical course is unpredictable, with pediatric cases faring better than adult ones, though visual impairment is common. Treatment remains debated, tailored to age, NF1 status, and tumor behavior, ranging from observation to surgery, chemotherapy, radiation, and targeted therapies like MAPK pathway inhibitors (e.g., for BRAF-altered tumors). Chemotherapy often delays radiation in young children to reduce cognitive risks, while targeted therapies are emerging for progressive cases. Despite treatment, long-term visual outcomes vary, and the lack of randomized trials underscores the need for further research to optimize management and enhance patient outcomes.