Ependymomas are central nervous system tumors that involve the ventricular system, and can develop during childhood. Diagnosis is based on MRI and biopsy. Treatment is a combination of surgery, radiation therapy, and sometimes chemotherapy.
Ependymomas are derived from the ependymal lining of the ventricular system and can occur in the supratentorial area, posterior fossa, or spine. Ependymomas are usually malignant.
Ependymomas are the third most common central nervous system (CNS) tumor in children (after astrocytomas and medulloblastomas), representing 5 to 10% of pediatric CNS tumors (1). Mean age at diagnosis is 5 to 8 years for ependymomas in the brain and is 12 years for ependymomas in the spine (2). Ependymomas also occur in infants.
This photo of a gross specimen shows a low-grade ependymoma appearing as a well-delimited nodule on the fornix beneath the septum pellucidum.
JOSE CALVO / SCIENCE PHOTO LIBRARY
In children, ependymomas most commonly occur in the posterior fossa, followed by the supratentorial area, and least commonly occur in the spine (1). In adults, however, ependymomas most commonly occur in the spine (3).
General references
1. Cohen AR. Brain Tumors in Children. N Engl J Med. 2022;386(20):1922-1931. doi:10.1056/NEJMra2116344
2. McGuire CS, Sainani KL, Fisher PG. Incidence patterns for ependymoma: A surveillance, epidemiology, and end results study. J Neurosurg. 2009;110(4):725–729. doi:10.3171/2008.9.JNS08117
3. Lampros M, Vlachos N, Alexiou GA. Ependymomas in Children and Adults. Adv Exp Med Biol. 2023;1405:99-116. doi:10.1007/978-3-031-23705-8_4
Symptoms and Signs of Ependymomas in Children
Initial presentation depends on the location of the tumor.
For supratentorial ependymomas, symptoms can include changes in personality or concentration, headaches, seizures, and focal neurologic deficits.
For posterior fossa tumors, symptoms are typically related to increased intracranial pressure. Infants may present with developmental delay, irritability, and increased head circumference. Older children typically present with nausea, vomiting, headache, ataxia/balance issues, and lethargy.
For spinal tumors, symptoms of spinal cord compression (eg, back pain, loss of bladder and bowel control) may occur.
Diagnosis of Ependymomas in Children
MRI
Histologic evaluation of biopsy specimen or entire resected tumor, with tumor molecular profiling
Diagnosis of ependymoma is based on MRI.
Definitive diagnosis is made using tumor tissue obtained by biopsy or ideally by gross total resection of the tumor at initial presentation. Molecular genetic profiling is used to classify ependymomas into different types.
Treatment of Ependymomas in Children
Surgical resection, usually followed by radiation therapy
Sometimes chemotherapy
Surgical resection is critical, and the degree of resection is one of the most important prognostic factors (1).
Radiation therapy has been shown to increase survival and should be given after surgery (1); however, a small subset of patients with supratentorial ependymomas who do not receive radiation can potentially be cured by surgery alone.
A study of 1100 children treated with proton therapy for intracranial ependymoma reported excellent outcomes: 5-year overall survival was 83%, progression-free survival was 74%, and late toxicities were modest (growth hormone deficiency occurred in 21%, hypothyroidism in 15%, radionecrosis in 2%, and secondary cancer in 0.65%). Subtotal resection was the sole predictor of inferior local control and progression-free survival (2).
Chemotherapy has not been clearly shown to improve survival (3), but, in some children, it may be used to shrink the tumor before gross total resection or a second-look surgery.
Treatment references
1. Lampros M, Vlachos N, Alexiou GA. Ependymomas in Children and Adults. Adv Exp Med Biol. 2023;1405:99-116. doi:10.1007/978-3-031-23705-8_4
2. Viani GA, Hamamura AC, Arruda CV, Cardoso CE, Salmon HA, Amaral GO. Proton radiotherapy outcomes in pediatric ependymoma: A long-term meta-analysis (PROPEL). Crit Rev Oncol Hematol. 2026;223:105203. doi:10.1016/j.critrevonc.2026.105203
3. Smith AA, Onar-Thomas A, Lensing S, et al. Phase 3 randomized trial of postirradiation chemotherapy in patients with newly diagnosed ependymoma: A report from the Children's Oncology Group. Neuro Oncol. 2026;28(5):1286-1297. doi:10.1093/neuonc/noaf285
Prognosis for Ependymomas in Children
The extent of surgical resection has a significant impact on survival rate (1−3):
Total or near-total resection: 61 to 70% overall survival
Subtotal resection: 32 to 37% overall survival
Children who survive are at risk of neurologic deficits resulting from the adverse effects of surgery and radiation therapy.
An advanced molecular–clinical risk stratification model for pediatric intracranial ependymoma based on a prospective trial and subsequent registry data incorporates molecular risk factors (1q gain, subtype PFA1c/d/e or 2a), tumor location, and extent of resection to distinguish between standard-, intermediate-, and high-risk tumors (4).
Prognosis references
1. Cage TA, Clark AJ, Aranda D, et al. A systematic review of treatment outcomes in pediatric patients with intracranial ependymomas. J Neurosurg Pediatr. 2013;11(6):673–681. doi:10.3171/2013.2.PEDS12345
2. Merchant TE, Bendel AE, Sabin ND, et al. Conformal Radiation Therapy for Pediatric Ependymoma, Chemotherapy for Incompletely Resected Ependymoma, and Observation for Completely Resected, Supratentorial Ependymoma. J Clin Oncol. 2019;37(12):974-983. doi:10.1200/JCO.18.01765
3. Snider CA, Yang K, Mack SC, et al. Impact of radiation therapy and extent of resection for ependymoma in young children: A population-based study. Pediatr Blood Cancer. 2018;65(3):10.1002/pbc.26880. doi:10.1002/pbc.26880
4. von Hoff K, Obrecht-Sturm D, Ghasemi DR, et al. Developing an advanced risk stratification model for pediatric intracranial ependymoma based on the prospective trial E-HIT2000 and subsequent registries. Neuro Oncol. 2026;28(2):520-534. doi:10.1093/neuonc/noaf218
