What is ependymoma?

An ependymoma is a rare type of brain cancer, mostly seen in children. The average age at diagnosis is 5 years. Nobody knows the cause of these tumors. There are approximately 150 cases diagnosed annually in the United States.

Ependymomas develop from the cells lining the ventricles, located deep within the brain. The ventricles are normally filled with cerebrospinal fluid (CSF), and help cushion the brain from trauma. When ependymomas develop, they often interfere with the flow of CSF within the brain, causing a build up of CSF – also known as hydrocephalus. This is often associated with headaches (or a rapidly increasing head circumference in infants) and is often the first symptom.

As with other brain tumors, imaging studies (such as a CT scan or MRI) are used to define the size and spread of a tumor and may suggest the diagnosis of ependymoma, but a surgical biopsy or resection is required to confirm the diagnosis.

What is the outcome?

The prognosis for children with ependymomas varies widely, depending on the characteristics of the tumor and other factors, including location and percentage of tumor removed by surgery. The percentage of tumor removed by the surgeon is the most predictive variable in ependymoma treatment. Patients whose tumors can be completely removed have a have much better prognosis than patients with incompletely removed ependymomas.

How serious is it?

Ependymoma is a serious condition that needs to be treated by a multi-disciplinary team consisting of neurosurgeons, neuro-oncologists and radiation oncologists. Even with appropriate therapies, a fraction of patients cannot be cured.

What are the treatment options?

As with most tumors, our treatments are limited to three modalities: surgery, radiation, and chemotherapy. Surgery is the most effective therapy for ependymomas, and is used whenever possible to treat ependymomas. Because of their deep location within the brain, it is often difficult to completely remove them without damaging vital brain structures. Depending on the tumor's location, your child's neurosurgeon may remove as much of the tumor as possible by creating an opening in the skull in an operation called a craniotomy. If the ependymoma is in a location where it can't be removed, surgery may be limited to a biopsy or partial removal of the tumor.

What clinical trials for ependymoma are currently enrolling patients?

There are generally several trials available for children with ependymoma as well as progressive/recurrent ependymoma. UCSF partners with the Pacific Pediatric Neuro-Oncology Consortium, the Children’s Oncology Group, and some industry sponsors to provide access to pediatric brain tumor clinical trials. UCSF is also part of CERN (Collaborative Ependymoma Research Effort), an organization with the mission to find new treatment options for patients with ependymoma.

Click here for a list of trials for pediatric brain tumors currently offered at UCSF. Or contact our pediatric oncology nurse practitioner line at (415)476-3831 for additional information.

What research is being done on ependymoma?

Ependymoma is an understudied disease. Current research is focusing on identifying genetic abnormalities in these tumors, and the generation of model systems of ependymoma in which new therapies can be tested.