Abstract
Survival rates for patients with ependymoma, a glial tumor arising from the ependymal cells lining the ventricles of the brain and spinal cord canal, have changed little during the past decade. Contemporary "standard" therapy for children and adults with ependymoma consists of maximal surgical resection followed by focal irradiation except in cases of disseminated disease. Despite refinements in radiotherapy techniques and improvements in survival for patients with gross totally resected, nonanaplastic disease, many therapeutic challenges remain, especially for patients with unresectable, macroscopic, metastatic, or anaplastic disease. Moreover, radiotherapy to the developing central nervous system, especially in patients younger than age 5 years, can have potential long-term neurocognitive and neuroendocrine sequelae. Chemotherapy has not played a role in most treatment regimens for ependymoma to date, but due to the ongoing therapeutic challenges for a subset of patients, this modality is being reinvestigated in a few ongoing studies. Early recognition of patients who will not respond to primary therapy is imperative to modify treatment regimens, such as intensification with the addition of adjuvant chemotherapy, the use of novel experimental therapies, or their combination. Refinements in patient stratification schemes that are based on a combination of clinical variables and molecular profiles also require improved knowledge of tumor biology. Several molecular alterations have been identified already, some of which may be of prognostic significance. Furthermore, disruption of molecular alterations in signaling pathways involved in the development and maintenance of ependymoma by using novel molecularly targeted therapies may improve outcomes and reduce toxicity for patients with ependymoma.
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