The authors provide an update on most issues related to biology, diagnosis, and treatment of children with ependymoma based on a literature review. Ependymoma is the third most common brain tumor in children and overall survival ranges from 24% to 75% at 5 years. The extent of surgical resection remains the principal risk factor that clearly influences outcome. The influence of age, location, grade, or stage has proved to be more controversial. Current standard therapy includes surgical resection and radiotherapy. Chemotherapy has a role in infants to avoid/delay radiotherapy and can be helpful to improve resectability. About half of patients will experience relapse, and outcome is dismal. New radiation modalities, reirradiation, chemotherapy, or targeted agents have been tested with promising results. Results of multi-institutional clinical trials are awaited to determine the best first-line treatment, while results of early phase I/ II trials will explore directed therapies based on new biologic factors.
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