Medulloblastoma is the most common malignant brain tumor of childhood, accounting for nearly 25% to 30% of primary central nervous system tumors in children younger than 18 years of age. Risk stratification into low and high risk categories has been based on age of clinical presentation, extent of postsurgical residual tumor, and disease dissemination. The World Health Organization (WHO) in 2007 recognized 5 histological subtypes as classic, anaplastic, large cell, desmoplastic/nodular, and medulloblastoma with extensive nodularity. Recent work with gene expression profiling along with histological classification has generated a novel combined histopathological and molecular stratification scheme into 4 subgroups (Wnt, Shh, group 3, and group 4). This could now help to identify patients who might benefit from dose escalation and de-escalation of therapy. Restratification brings optimism in treating these patients as scholars now have the ability to profile a more targeted therapy approach. This review discusses the literature regarding this new research endeavor.

MEK inhibitors (MEKi) represent innovative and promising treatments for managing manifestations of neurofibromatosis type 1 (NF1). To mitigate potential ophthalmic side effects, such as MEKi-associated retinopathy (MEKAR), patients undergoing MEKi therapy routinely receive ophthalmology evaluations. Our study aims to assess the necessity of this regular screening within a predominantly pediatric NF1 population by examining the occurrence of ocular adverse events (OAE). A retrospective study evaluated 45 NF1 patients receiving MEKi. Inclusion criteria included baseline and follow-up examinations following the initiation of MEKi therapy. At each assessment, a comprehensive eye evaluation was performed, comprising a dilated fundus examination, ocular coherence tomography of the macula and nerve fiber layer, and Humphrey visual field testing. Twenty-six patients, with an average age of 13 years (range 2–23 years) and an average follow-up duration of 413 days were included in the analysis. Three different MEKi were used: selumetinib (77%), trametinib (23%), and mirdametinib (4%). None of the patients experienced retinopathy at any point during the study. Some patients had pre-existing optic neuropathies (27%), but no instances of nerve changes occurred after commencing MEKi therapy. Four patients (15%) exhibited symptoms of dry eye, all of which were effectively managed with topical lubrication.