Purpose of review: This article describes the most common pediatric brain tumors and highlights recent developments in their diagnosis and treatment strategies.
Recent findings: We are in the midst of a molecular era for pediatric brain tumors. Genetic and epigenetic profiling of tumors has impacted their diagnosis, allowing for the subgrouping of heterogeneous tumor groups and leading to the complete renaming of some tumor types. These advances are reflected in the new 2016 World Health Organization classification. For example, primitive neuroectodermal tumors have been completely eliminated and replaced by subgroups defined by the absence or presence of specific chromosomal amplification. Medulloblastomas, diffuse astrocytomas, and ependymomas now have specific subtypes that are based on defining molecular features. More recent epigenetic-based subgrouping of atypical teratoid/rhabdoid tumors have not yet made it into the official classification system, but will surely have an impact on how these tumors are regarded in future preclinical and clinical trials.
Summary: Genetic and epigenetic data are changing how pediatric brain tumors are diagnosed, are leading to new guidelines for how treatment outcome analyses can be organized, and are offering molecular targets that can be used for the development of novel therapies.