Purpose of This PDQ Summary
General Information

Incidence and Molecular Determinants

Histopathologic Classification of Childhood Ependymoma
Stage Information
Treatment Option Overview
Treatment of Newly Diagnosed Childhood Ependymoma

Postsurgical Treatment Options         Standard treatment options         Treatment options under clinical evaluation Current Clinical Trials

Treatment of Recurrent Childhood Ependymoma

Current Clinical Trials

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Changes to This Summary (11/26/2008)
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Purpose of This PDQ Summary

This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of childhood ependymoma. This summary is reviewed regularly and updated as necessary by the PDQ Pediatric Treatment Editorial Board.

Information about the following is included in this summary:

• Histopathologic classification.
• Stage information.
• Treatment options.

This summary is intended as a resource to inform and assist clinicians and other health professionals who care for pediatric cancer patients. It does not provide formal guidelines or recommendations for making health care decisions.

Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Pediatric and Adult Treatment Editorial Boards use a formal evidence ranking system in developing their level-of-evidence designations. Based on the strength of the available evidence, treatment options are described as either “standard” or “under clinical evaluation.” These classifications should not be used as a basis for reimbursement determinations.

This summary is also available in a patient version, which is written in less-technical language, and in Spanish.  [Note: The PDQ childhood brain tumor treatment summaries are in the process of being substantially revised. This revision process was prompted by changes in the nomenclature and classification for pediatric central nervous system tumors. New PDQ childhood brain tumor treatment summaries will be added and some existing summaries will be replaced or their content combined with other PDQ childhood brain tumor treatment summaries in the near future.]

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General Information

The National Cancer Institute provides the PDQ pediatric cancer treatment information summaries as a public service to increase the availability of evidence-based cancer information to health professionals, patients, and the public.

In recent decades, dramatic improvements in survival have been achieved for children and adolescents with cancer. Childhood and adolescent cancer survivors require close follow-up because cancer therapy side effects may persist or develop months or years after treatment. (Refer to the PDQ summary Late Effects of Treatment for Childhood Cancer for specific information about the incidence, type, and monitoring of late effects in childhood and adolescent cancer survivors.)

Primary brain tumors are a diverse group of diseases that together constitute the most common solid tumor of childhood. Brain tumors are classified according to histology, but tumor location and extent of spread are important factors that affect treatment and prognosis. Immunohistochemical analysis, cytogenetic and molecular genetic findings, and measures of mitotic activity are increasingly used in tumor diagnosis and classification.

Refer to the PDQ summary Childhood Brain and Spinal Cord Tumors Treatment Overview for information about the general classification of childhood brain and spinal cord tumors.

Note: Some citations in the text of this section are followed by a level of evidence. The PDQ editorial boards use a formal ranking system to help the reader judge the strength of evidence linked to the reported results of a therapeutic strategy. (Refer to the PDQ summary Levels of Evidence for more information.)

Childhood ependymoma comprises approximately 9% of all childhood brain tumors representing approximately 200 cases per year in the United States.[1,2]

Molecular determinants of outcome for ependymomas are just being identified. Studies have identified numerous chromosomal aberrations and related molecular genetic changes. Gain of 1q25, overexpression of EGFR, hTERT expression , and others have been related to poorer prognosis.[3-5][Level of evidence: 3iiiB]

References

1. Gurney JG, Smith MA, Bunin GR: CNS and miscellaneous intracranial and intraspinal neoplasms. In: Ries LA, Smith MA, Gurney JG, et al., eds.: Cancer incidence and survival among children and adolescents: United States SEER Program 1975-1995. Bethesda, Md: National Cancer Institute, SEER Program, 1999. NIH Pub.No. 99-4649., Chapter 3, pp 51-63. Also available online. Last accessed March 14, 2007. 
   
   
2. Central Brain Tumor Registry of the United States.: Statistical Report: Primary Brain Tumors in the United States, 1997-2001. Hinsdale, Ill: Central Brain Tumor Registry of the United States, 2004. Also available online. Last accessed July 20, 2006. 
   
   
3. Tabori U, Ma J, Carter M, et al.: Human telomere reverse transcriptase expression predicts progression and survival in pediatric intracranial ependymoma. J Clin Oncol 24 (10): 1522-8, 2006.  [PUBMED Abstract]
   
   
4. Mendrzyk F, Korshunov A, Benner A, et al.: Identification of gains on 1q and epidermal growth factor receptor overexpression as independent prognostic markers in intracranial ependymoma. Clin Cancer Res 12 (7 Pt 1): 2070-9, 2006.  [PUBMED Abstract]
   
   
5. Pezzolo A, Capra V, Raso A, et al.: Identification of novel chromosomal abnormalities and prognostic cytogenetics markers in intracranial pediatric ependymoma. Cancer Lett 261 (2): 235-43, 2008.  [PUBMED Abstract]
   
   

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Histopathologic Classification of Childhood Ependymoma

The classification of brain tumors is based on both histopathological characteristics and location in the brain. Ependymomas are divided into the following categories:

• Subependymoma (World Health Organization [WHO] Grade I).



• Myxopapillary ependymoma (WHO Grade I).



• Ependymoma (WHO Grade II). Variants include cellular, papillary, tanycytic, clear cell, and mixed.



• Anaplastic (also known as malignant) ependymoma (WHO Grade III).

The most recent WHO classification of brain tumors maintains the term “ependymoma” for tumors that are histologically benign and malignant ependymoma for those that have malignant characteristics.[1] These categories are based on the nuclear/cytoplasmic ratio, number of nuclei and mitotic figures, and the degree of nuclear atypia. Contemporary studies have failed to show significant differences in how these tumors behave on the basis of histologic classification alone,[2-5] although a small experience from a single-institution study suggested that patients with clear cell ependymoma may be at higher risk for treatment failure,[6] confirmation is required in a larger group of unselected patients. Myxopapillary ependymomas, which typically present in the filum terminale and cauda equina, are also considered a separate entity. Ependymoblastomas (for more information, refer to the PDQ summary Childhood Central Nervous System Embryonal Tumors), which generally behave more like medulloblastomas or cortical neuroectodermal tumors, are considered separate entities from ependymomas and are now classified with the embryonal tumors.[1]

The pathologic classification of pediatric brain tumors is a specialized area that is undergoing evolution; review of the diagnostic tissue by a neuropathologist who has particular expertise in this area is strongly recommended.

References

1. Kleihues P, Burger PC, Scheithauer BW: The new WHO classification of brain tumours. Brain Pathol 3 (3): 255-68, 1993.  [PUBMED Abstract]
   
   
2. Goldwein JW, Leahy JM, Packer RJ, et al.: Intracranial ependymomas in children. Int J Radiat Oncol Biol Phys 19 (6): 1497-502, 1990.  [PUBMED Abstract]
   
   
3. Rousseau P, Habrand JL, Sarrazin D, et al.: Treatment of intracranial ependymomas of children: review of a 15-year experience. Int J Radiat Oncol Biol Phys 28 (2): 381-6, 1994.  [PUBMED Abstract]
   
   
4. Chiu JK, Woo SY, Ater J, et al.: Intracranial ependymoma in children: analysis of prognostic factors. J Neurooncol 13 (3): 283-90, 1992.  [PUBMED Abstract]
   
   
5. Pollack IF, Gerszten PC, Martinez AJ, et al.: Intracranial ependymomas of childhood: long-term outcome and prognostic factors. Neurosurgery 37 (4): 655-66; discussion 666-7, 1995.  [PUBMED Abstract]
   
   
6. Fouladi M, Helton K, Dalton J, et al.: Clear cell ependymoma: a clinicopathologic and radiographic analysis of 10 patients. Cancer 98 (10): 2232-44, 2003.  [PUBMED Abstract]
   
   

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Stage Information

Note: Some citations in the text of this section are followed by a level of evidence. The PDQ editorial boards use a formal ranking system to help the reader judge the strength of evidence linked to the reported results of a therapeutic strategy. (Refer to the PDQ summary Levels of Evidence for more information.)

Although there is no formal staging system, ependymomas can be divided into supratentorial and infratentorial tumors. They usually originate in the ependymal linings of ventricles in the posterior fossa or supratentorial region, and have access to the cerebral spinal fluid (CSF) and therefore may spread throughout the entire neuraxis. Thirty percent of childhood ependymomas arise outside of the posterior fossa.[1-3] Every patient with ependymoma should be evaluated with diagnostic imaging of the spinal cord and whole brain. The most sensitive method available for evaluating spinal cord subarachnoid metastasis is spinal magnetic resonance imaging (MRI) performed with gadolinium. If MRI is used, the entire spine is generally imaged in at least two planes with contiguous MRI slices performed after gadolinium enhancement. In addition, CSF cytological evaluation should be conducted. While a number of factors have sometimes been associated with an unfavorable outcome (younger age at diagnosis, lower doses of radiation, anaplastic histology, subtotal resection, higher proliferation marker, MIB-1 labeling index),[1,4-10][Level of evidence: 3iiiDi] age, histology, and extent of resection have consistently been the most important factors.[5,6,11,12] Molecular diagnostics are evolving, but have yet to be validated in a prospective manner.[13,14] These prognostic variables must be evaluated in the context of the treatment received.

References

1. Goldwein JW, Leahy JM, Packer RJ, et al.: Intracranial ependymomas in children. Int J Radiat Oncol Biol Phys 19 (6): 1497-502, 1990.  [PUBMED Abstract]
   
   
2. Kovnar E, Kun L, Burger J, et al.: Patterns of dissemination and recurrence in childhood ependymoma: preliminary results of Pediatric Oncology Group protocol #8532. Ann Neurol 30(3): 457, 1991. 
   
   
3. Vanuytsel LJ, Bessell EM, Ashley SE, et al.: Intracranial ependymoma: long-term results of a policy of surgery and radiotherapy. Int J Radiat Oncol Biol Phys 23 (2): 313-9, 1992.  [PUBMED Abstract]
   
   
4. Shaw EG, Evans RG, Scheithauer BW, et al.: Postoperative radiotherapy of intracranial ependymoma in pediatric and adult patients. Int J Radiat Oncol Biol Phys 13 (10): 1457-62, 1987.  [PUBMED Abstract]
   
   
5. Horn B, Heideman R, Geyer R, et al.: A multi-institutional retrospective study of intracranial ependymoma in children: identification of risk factors. J Pediatr Hematol Oncol 21 (3): 203-11, 1999 May-Jun.  [PUBMED Abstract]
   
   
6. Pollack IF, Gerszten PC, Martinez AJ, et al.: Intracranial ependymomas of childhood: long-term outcome and prognostic factors. Neurosurgery 37 (4): 655-66; discussion 666-7, 1995.  [PUBMED Abstract]
   
   
7. Merchant TE, Jenkins JJ, Burger PC, et al.: Influence of tumor grade on time to progression after irradiation for localized ependymoma in children. Int J Radiat Oncol Biol Phys 53 (1): 52-7, 2002.  [PUBMED Abstract]
   
   
8. Wolfsberger S, Fischer I, Höftberger R, et al.: Ki-67 immunolabeling index is an accurate predictor of outcome in patients with intracranial ependymoma. Am J Surg Pathol 28 (7): 914-20, 2004.  [PUBMED Abstract]
   
   
9. Kurt E, Zheng PP, Hop WC, et al.: Identification of relevant prognostic histopathologic features in 69 intracranial ependymomas, excluding myxopapillary ependymomas and subependymomas. Cancer 106 (2): 388-95, 2006.  [PUBMED Abstract]
   
   
10. Tihan T, Zhou T, Holmes E, et al.: The prognostic value of histological grading of posterior fossa ependymomas in children: a Children's Oncology Group study and a review of prognostic factors. Mod Pathol 21 (2): 165-77, 2008.  [PUBMED Abstract]
    
    
11. Bouffet E, Perilongo G, Canete A, et al.: Intracranial ependymomas in children: a critical review of prognostic factors and a plea for cooperation. Med Pediatr Oncol 30 (6): 319-29; discussion 329-31, 1998.  [PUBMED Abstract]
    
    
12. Korshunov A, Golanov A, Sycheva R, et al.: The histologic grade is a main prognostic factor for patients with intracranial ependymomas treated in the microneurosurgical era: an analysis of 258 patients. Cancer 100 (6): 1230-7, 2004.  [PUBMED Abstract]
    
    
13. Mendrzyk F, Korshunov A, Benner A, et al.: Identification of gains on 1q and epidermal growth factor receptor overexpression as independent prognostic markers in intracranial ependymoma. Clin Cancer Res 12 (7 Pt 1): 2070-9, 2006.  [PUBMED Abstract]
    
    
14. Tabori U, Ma J, Carter M, et al.: Human telomere reverse transcriptase expression predicts progression and survival in pediatric intracranial ependymoma. J Clin Oncol 24 (10): 1522-8, 2006.  [PUBMED Abstract]
    
    

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Treatment Option Overview

Note: Some citations in the text of this section are followed by a level of evidence. The PDQ editorial boards use a formal ranking system to help the reader judge the strength of evidence linked to the reported results of a therapeutic strategy. (Refer to the PDQ summary Levels of Evidence for more information.)

Many of the improvements in survival in childhood cancer have been made as a result of clinical trials that have attempted to improve on the best available, accepted therapy. Clinical trials in pediatrics are designed to compare new therapy with therapy that is currently accepted as standard. This comparison may be done in a randomized study of two treatment arms or by evaluating a single new treatment and comparing the results with those previously obtained with existing therapy.

Because of the relative rarity of cancer in children, all patients with brain tumors should be considered for entry into a clinical trial. To determine and implement optimum treatment, treatment planning by a multidisciplinary team of cancer specialists who have experience treating childhood brain tumors is required. Radiation therapy of pediatric brain tumors is technically very demanding and should be carried out in centers that have experience in that area in order to ensure optimal results.

In the past, treatment for childhood ependymoma has included surgery with radiation therapy. There is evidence to suggest that more extensive surgical resections are related to an improved rate of survival.[1-6] Chemotherapy has been shown to be active in patients with recurrent ependymoma.[7] One relatively small, prospective, randomized trial suggests that chemotherapy activity in newly diagnosed cases is limited,[8] and current treatment approaches do not include chemotherapy as a component of primary therapy for most children with newly diagnosed ependymomas that are completely resected. Children younger than 3 years are particularly susceptible to the adverse effect of radiation on brain development.[9][Level of evidence: 3iiiC] Debilitating effects on growth and neurologic development have frequently been observed, especially in younger children.[10-12] For this reason, conformal radiation approaches that minimize damage to normal brain tissue are under evaluation for infants and children with ependymoma.[13] Long-term management of these patients is complex and requires a multidisciplinary approach.

There is evidence that surveillance neuroimaging in childhood ependymoma will identify tumors that have recurred when the patient is asymptomatic; however, it is unclear whether this detection will change the ultimate prognosis of the patient.[14]

References

1. Pollack IF, Gerszten PC, Martinez AJ, et al.: Intracranial ependymomas of childhood: long-term outcome and prognostic factors. Neurosurgery 37 (4): 655-66; discussion 666-7, 1995.  [PUBMED Abstract]
   
   
2. Horn B, Heideman R, Geyer R, et al.: A multi-institutional retrospective study of intracranial ependymoma in children: identification of risk factors. J Pediatr Hematol Oncol 21 (3): 203-11, 1999 May-Jun.  [PUBMED Abstract]
   
   
3. van Veelen-Vincent ML, Pierre-Kahn A, Kalifa C, et al.: Ependymoma in childhood: prognostic factors, extent of surgery, and adjuvant therapy. J Neurosurg 97 (4): 827-35, 2002.  [PUBMED Abstract]
   
   
4. Abdel-Wahab M, Etuk B, Palermo J, et al.: Spinal cord gliomas: A multi-institutional retrospective analysis. Int J Radiat Oncol Biol Phys 64 (4): 1060-71, 2006.  [PUBMED Abstract]
   
   
5. Kothbauer KF: Neurosurgical management of intramedullary spinal cord tumors in children. Pediatr Neurosurg 43 (3): 222-35, 2007.  [PUBMED Abstract]
   
   
6. Zacharoulis S, Ji L, Pollack IF, et al.: Metastatic ependymoma: a multi-institutional retrospective analysis of prognostic factors. Pediatr Blood Cancer 50 (2): 231-5, 2008.  [PUBMED Abstract]
   
   
7. Goldwein JW, Glauser TA, Packer RJ, et al.: Recurrent intracranial ependymomas in children. Survival, patterns of failure, and prognostic factors. Cancer 66 (3): 557-63, 1990.  [PUBMED Abstract]
   
   
8. Evans AE, Anderson JR, Lefkowitz-Boudreaux IB, et al.: Adjuvant chemotherapy of childhood posterior fossa ependymoma: cranio-spinal irradiation with or without adjuvant CCNU, vincristine, and prednisone: a Childrens Cancer Group study. Med Pediatr Oncol 27 (1): 8-14, 1996.  [PUBMED Abstract]
   
   
9. von Hoff K, Kieffer V, Habrand JL, et al.: Impairment of intellectual functions after surgery and posterior fossa irradiation in children with ependymoma is related to age and neurologic complications. BMC Cancer 8: 15, 2008.  [PUBMED Abstract]
   
   
10. Packer RJ, Sutton LN, Atkins TE, et al.: A prospective study of cognitive function in children receiving whole-brain radiotherapy and chemotherapy: 2-year results. J Neurosurg 70 (5): 707-13, 1989.  [PUBMED Abstract]
    
    
11. Johnson DL, McCabe MA, Nicholson HS, et al.: Quality of long-term survival in young children with medulloblastoma. J Neurosurg 80 (6): 1004-10, 1994.  [PUBMED Abstract]
    
    
12. Packer RJ, Sutton LN, Goldwein JW, et al.: Improved survival with the use of adjuvant chemotherapy in the treatment of medulloblastoma. J Neurosurg 74 (3): 433-40, 1991.  [PUBMED Abstract]
    
    
13. Merchant TE, Mulhern RK, Krasin MJ, et al.: Preliminary results from a phase II trial of conformal radiation therapy and evaluation of radiation-related CNS effects for pediatric patients with localized ependymoma. J Clin Oncol 22 (15): 3156-62, 2004.  [PUBMED Abstract]
    
    
14. Good CD, Wade AM, Hayward RD, et al.: Surveillance neuroimaging in childhood intracranial ependymoma: how effective, how often, and for how long? J Neurosurg 94 (1): 27-32, 2001.  [PUBMED Abstract]
    
    

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Treatment of Newly Diagnosed Childhood Ependymoma

Note: Some citations in the text of this section are followed by a level of evidence. The PDQ editorial boards use a formal ranking system to help the reader judge the strength of evidence linked to the reported results of a therapeutic strategy. (Refer to the PDQ summary Levels of Evidence for more information.)

In the newly diagnosed patient, careful evaluation to fully determine the extent of disease must precede the treatment of ependymoma. Surgery should be performed in an attempt at maximal tumor reduction; children have improved progression-free survival (PFS) if there is minimal residual disease present after surgery.[1,2] Postoperatively, magnetic resonance imaging (MRI) should be performed to determine the extent of resection. If not performed preoperatively, MRI of the entire neuraxis should be obtained to evaluate for disease dissemination. Even myxopapillary ependymomas, considered to be a somewhat benign histologic subtype of ependymoma, have a relatively high incidence of central nervous system (CNS) tumor dissemination at diagnosis and at follow-up, thus underscoring the need for imaging of the complete cranial spinal axis at the time of diagnosis and during follow-up.[3,4] Patients with residual tumor or disseminated disease should be considered at high risk for relapse and should be treated on protocols specifically designed for them. Those with no evidence of residual tumor still have an approximate 20% to 40% relapse risk in spite of postoperative radiation therapy.

Ependymoma (World Health Organization [WHO] Grade II) and anaplastic (WHO Grade III)

• No residual disease; no disseminated disease:

  The traditional postsurgical treatment for these patients has been radiation therapy consisting of 54 Gy to 55.8 Gy to the tumor bed for children aged 3 years and older and is under evaluation for children younger than 3 years. It is not necessary to treat the entire CNS (whole brain and spine) because these tumors usually recur at the local site.[2,5] When possible, patients should be treated in a center experienced with this therapy. There is no evidence that adjuvant chemotherapy, including the use of myeloablative chemotherapy,[6] improves the outcome for patients with totally resected, nondisseminated ependymoma. The 3-year PFS rate in 74 patients aged between 1 and 21 years treated with radiation therapy following surgery was 77.6% ± 5.8%.[7] Limited experience suggests that surgery alone for completely resected supratentorial nonanaplastic tumors,[8] and intradural spinal cord ependymomas [9][Level of evidence: 3iiiDi] may, in select cases, be an appropriate approach to treatment.




• Residual disease; no disseminated disease:

  Second-look surgery should be considered because patients who have complete resections have better disease control. The traditional postsurgical treatment for children aged 3 years and older has been radiation therapy consisting of 54 Gy to 55.8 Gy to the tumor bed. It is not necessary to treat the entire CNS (whole brain and spine) because these tumors usually recur at the local site. In subtotally resected patients, treatment with radiation therapy results in 3-year to 5-year PFS in 30% to 50% of patients,[7,10] although the outcome for patients with residual tumor within the spinal canal may be better.[11] There is no evidence that adjuvant chemotherapy, including high-dose chemotherapy with stem cell rescue, is of any benefit.[12]




• CNS disseminated disease:

  In children with disseminated disease, long-term survivors have been reported and aggressive therapy is warranted. Regardless of degree of surgical resection, these patients require radiation therapy to the entire CNS (whole brain and spine) along with boosts to local disease and bulk areas of disseminated disease. The traditional local postsurgical radiation doses in these patients have been 54 Gy to 55.8 Gy. Doses of approximately 36 Gy to the entire neuraxis (i.e., the whole brain and spine) should also be administered, but may be modulated depending on the age of the patient. Boosts between 41.4 Gy and 50.4 Gy to bulk areas of spinal disease should be administered, with doses depending on the age of the patient and the location of the tumor. When possible, patients should be treated in a center experienced with this therapy. Trials are ongoing to evaluate the possible role of radiation therapy and chemotherapy in these patients.




• Management of children younger than 3 years:

  Because of the known effects of radiation on growth and neurocognitive development, radiation therapy immediately after surgery in children younger than 3 years has traditionally been limited, with attempts to delay its administration through the use of chemotherapy.[13-16] When analyzing neurologic outcome following treatment of young children with ependymoma, it is important to consider that not all long-term deficits can be ascribed to radiation therapy, as deficits may be present in young children before therapy is begun.[7] For example, the presence of hydrocephalus at diagnosis is associated with lower intelligence quotient as measured following surgical resection and prior to administration of radiation therapy.[17]

  Chemotherapy is able to induce objective responses in some children younger than 3 years with newly diagnosed ependymoma,[13-15] though not all chemotherapy regimens induce objective responses.[16] Up to 40% of infants and young children with totally resected disease may achieve long-term survival with chemotherapy alone.[18][Level of evidence: 2Di] The need and timing of radiation therapy for children who have successfully completed chemotherapy and have no residual disease is still to be determined. Current approaches to treating young children with ependymoma do not generally employ chemotherapy to delay use of radiation therapy.

  Conformal radiation therapy is an alternative approach for minimizing radiation-induced neurologic damage in young children with ependymoma. The initial experience with this approach suggests that children younger than 3 years with ependymoma have neurologic deficits at diagnosis that improve with time following conformal radiation treatment.[7] The current Children's Oncology Group (COG) protocol for children with ependymoma includes young children aged 12 to 36 months and is evaluating whether conformal radiation therapy can minimize neurologic late effects while producing long-term survival.

Myxopapillary ependymoma

• Historically, the management of myxopapillary ependymoma consisted of an attempt at en bloc resection of the tumor with no further treatment in the case of a gross total resection. However, based on the finding that dissemination of these tumors to other parts of the neuraxis can occur,[3] and evidence that focal irradiation may improve progression-free survival,[19] many practitioners now favor the use of irradiation following surgical resection of the primary mass.

The following is an example of a national and/or institutional clinical trial that is currently being conducted. Information about ongoing clinical trials is available from the NCI Web site.

A COG trial (COG-ACNSO121) is now closed to accrual and analysis is pending. The study evaluated several questions of therapy for different subgroups of children aged 1 year and older with ependymoma as described below:

No Residual Disease; No Disseminated Disease:

• Children who have supratentorial nonanaplastic ependymoma for whom a gross total resection can be performed: These children are being carefully observed following surgical resection to determine whether they can be cured with surgery alone.



• Children with supratentorial anaplastic ependymoma and children with infratentorial ependymoma who have a near total resection or better: These children receive conformal radiation therapy directed at the primary site to determine whether cure can be achieved with this approach while minimizing radiation-associated long-term toxicities. Children with supratentorial nonanaplastic ependymoma with a near–total resection or better but who are not eligible for the observation also receive conformal radiation.

Residual Disease; No Disseminated Disease:

• Children with initial incompletely resected ependymoma: These children receive chemotherapy in an attempt to achieve a complete resection with second surgery prior to conformal radiation therapy.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with newly diagnosed childhood ependymoma. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.

General information about clinical trials is also available from the NCI Web site.

References

1. Hukin J, Epstein F, Lefton D, et al.: Treatment of intracranial ependymoma by surgery alone. Pediatr Neurosurg 29 (1): 40-5, 1998.  [PUBMED Abstract]
   
   
2. Horn B, Heideman R, Geyer R, et al.: A multi-institutional retrospective study of intracranial ependymoma in children: identification of risk factors. J Pediatr Hematol Oncol 21 (3): 203-11, 1999 May-Jun.  [PUBMED Abstract]
   
   
3. Fassett DR, Pingree J, Kestle JR: The high incidence of tumor dissemination in myxopapillary ependymoma in pediatric patients. Report of five cases and review of the literature. J Neurosurg 102 (1 Suppl): 59-64, 2005.  [PUBMED Abstract]
   
   
4. Bagley CA, Kothbauer KF, Wilson S, et al.: Resection of myxopapillary ependymomas in children. J Neurosurg 106 (4 Suppl): 261-7, 2007.  [PUBMED Abstract]
   
   
5. Evans AE, Anderson JR, Lefkowitz-Boudreaux IB, et al.: Adjuvant chemotherapy of childhood posterior fossa ependymoma: cranio-spinal irradiation with or without adjuvant CCNU, vincristine, and prednisone: a Childrens Cancer Group study. Med Pediatr Oncol 27 (1): 8-14, 1996.  [PUBMED Abstract]
   
   
6. Zacharoulis S, Levy A, Chi SN, et al.: Outcome for young children newly diagnosed with ependymoma, treated with intensive induction chemotherapy followed by myeloablative chemotherapy and autologous stem cell rescue. Pediatr Blood Cancer 49 (1): 34-40, 2007.  [PUBMED Abstract]
   
   
7. Merchant TE, Mulhern RK, Krasin MJ, et al.: Preliminary results from a phase II trial of conformal radiation therapy and evaluation of radiation-related CNS effects for pediatric patients with localized ependymoma. J Clin Oncol 22 (15): 3156-62, 2004.  [PUBMED Abstract]
   
   
8. Goldwein JW, Leahy JM, Packer RJ, et al.: Intracranial ependymomas in children. Int J Radiat Oncol Biol Phys 19 (6): 1497-502, 1990.  [PUBMED Abstract]
   
   
9. Volpp PB, Han K, Kagan AR, et al.: Outcomes in treatment for intradural spinal cord ependymomas. Int J Radiat Oncol Biol Phys 69 (4): 1199-204, 2007.  [PUBMED Abstract]
   
   
10. Pollack IF, Gerszten PC, Martinez AJ, et al.: Intracranial ependymomas of childhood: long-term outcome and prognostic factors. Neurosurgery 37 (4): 655-66; discussion 666-7, 1995.  [PUBMED Abstract]
    
    
11. Wahab SH, Simpson JR, Michalski JM, et al.: Long term outcome with post-operative radiation therapy for spinal canal ependymoma. J Neurooncol 83 (1): 85-9, 2007.  [PUBMED Abstract]
    
    
12. Grill J, Kalifa C, Doz F, et al.: A high-dose busulfan-thiotepa combination followed by autologous bone marrow transplantation in childhood recurrent ependymoma. A phase-II study. Pediatr Neurosurg 25 (1): 7-12, 1996.  [PUBMED Abstract]
    
    
13. Duffner PK, Horowitz ME, Krischer JP, et al.: The treatment of malignant brain tumors in infants and very young children: an update of the Pediatric Oncology Group experience. Neuro-oncol 1 (2): 152-61, 1999.  [PUBMED Abstract]
    
    
14. Duffner PK, Horowitz ME, Krischer JP, et al.: Postoperative chemotherapy and delayed radiation in children less than three years of age with malignant brain tumors. N Engl J Med 328 (24): 1725-31, 1993.  [PUBMED Abstract]
    
    
15. Geyer JR, Sposto R, Jennings M, et al.: Multiagent chemotherapy and deferred radiotherapy in infants with malignant brain tumors: a report from the Children's Cancer Group. J Clin Oncol 23 (30): 7621-31, 2005.  [PUBMED Abstract]
    
    
16. Grill J, Le Deley MC, Gambarelli D, et al.: Postoperative chemotherapy without irradiation for ependymoma in children under 5 years of age: a multicenter trial of the French Society of Pediatric Oncology. J Clin Oncol 19 (5): 1288-96, 2001.  [PUBMED Abstract]
    
    
17. Merchant TE, Lee H, Zhu J, et al.: The effects of hydrocephalus on intelligence quotient in children with localized infratentorial ependymoma before and after focal radiation therapy. J Neurosurg 101 (2 Suppl): 159-68, 2004.  [PUBMED Abstract]
    
    
18. Grundy RG, Wilne SA, Weston CL, et al.: Primary postoperative chemotherapy without radiotherapy for intracranial ependymoma in children: the UKCCSG/SIOP prospective study. Lancet Oncol 8 (8): 696-705, 2007.  [PUBMED Abstract]
    
    
19. Akyurek S, Chang EL, Yu TK, et al.: Spinal myxopapillary ependymoma outcomes in patients treated with surgery and radiotherapy at M.D. Anderson Cancer Center. J Neurooncol 80 (2): 177-83, 2006.  [PUBMED Abstract]
    
    

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Treatment of Recurrent Childhood Ependymoma

Recurrence is not uncommon in both benign and malignant childhood brain tumors and may develop many years after initial treatment. For ependymoma, delays beyond 10 to 15 years have been reported.[1,2] Disease generally recurs at the primary tumor site, even in children with malignant ependymomas.[3,4] Systemic relapse is extremely rare. At time of relapse, a complete evaluation for extent of recurrence is indicated for all patients. The need for surgical intervention must be individualized on the basis of the extent of tumor, the length of time between initial treatment and the reappearance of the recurrent lesion, and the clinical picture. Patients with recurrent ependymomas who have not previously received radiation therapy and/or chemotherapy should be considered for treatment with these modalities. In addition, patients may be candidates for focal retreatment with various radiation modalities, including stereotactic radiosurgery. Active agents include cyclophosphamide, cisplatin, carboplatin, lomustine, and etoposide. Entry into studies of novel therapeutic approaches should be considered. Information about ongoing clinical trials is available from the NCI Web site.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with recurrent childhood ependymoma. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.

General information about clinical trials is also available from the NCI Web site.

References

1. Pollack IF, Gerszten PC, Martinez AJ, et al.: Intracranial ependymomas of childhood: long-term outcome and prognostic factors. Neurosurgery 37 (4): 655-66; discussion 666-7, 1995.  [PUBMED Abstract]
   
   
2. Vanuytsel LJ, Bessell EM, Ashley SE, et al.: Intracranial ependymoma: long-term results of a policy of surgery and radiotherapy. Int J Radiat Oncol Biol Phys 23 (2): 313-9, 1992.  [PUBMED Abstract]
   
   
3. Goldwein JW, Corn BW, Finlay JL, et al.: Is craniospinal irradiation required to cure children with malignant (anaplastic) intracranial ependymomas? Cancer 67 (11): 2766-71, 1991.  [PUBMED Abstract]
   
   
4. Merchant TE, Haida T, Wang MH, et al.: Anaplastic ependymoma: treatment of pediatric patients with or without craniospinal radiation therapy. J Neurosurg 86 (6): 943-9, 1997.  [PUBMED Abstract]
   
   

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Changes to This Summary (11/26/2008)

The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.

General Information

Added text to state that some reference citations are accompanied by a level-of-evidence designation that is intended to help readers assess the strength of evidence supporting specific interventions or approaches.

Added Pezzolo et al. as reference 5 and level of evidence 3iiiB.

Stage Information

Added text to state that some reference citations are accompanied by a level-of-evidence designation that is intended to help readers assess the strength of evidence supporting specific interventions or approaches.

Added Tihan et al. as reference 10 and level of evidence 3iiiDi.

Treatment Option Overview

Added text to state that some reference citations are accompanied by a level-of-evidence designation that is intended to help readers assess the strength of evidence supporting specific interventions or approaches.

Added von Hoff et al. as reference 9 and level of evidence 3iiiC.

Treatment of Newly Diagnosed Childhood Ependymoma

Added text to state that up to 40% of infants and young children with totally resected disease may achieve long-term survival with chemotherapy alone (cited Grundy et al. as reference 18 and level of evidence 2Di).

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More Information

About PDQ

• PDQ® - NCI's Comprehensive Cancer Database.

  Full description of the NCI PDQ database.

Additional PDQ Summaries

• PDQ® Cancer Information Summaries: Adult Treatment

  Treatment options for adult cancers.

• PDQ® Cancer Information Summaries: Pediatric Treatment

  Treatment options for childhood cancers.

• PDQ® Cancer Information Summaries: Supportive and Palliative Care

  Side effects of cancer treatment, management of cancer-related complications and pain, and psychosocial concerns.

• PDQ® Cancer Information Summaries: Screening/Detection (Testing for Cancer)

  Tests or procedures that detect specific types of cancer.

• PDQ® Cancer Information Summaries: Prevention

  Risk factors and methods to increase chances of preventing specific types of cancer.

• PDQ® Cancer Information Summaries: Genetics

  Genetics of specific cancers and inherited cancer syndromes, and ethical, legal, and social concerns.

• PDQ® Cancer Information Summaries: Complementary and Alternative Medicine

  Information about complementary and alternative forms of treatment for patients with cancer.

Important:

This information is intended mainly for use by doctors and other health care professionals. If you have questions about this topic, you can ask your doctor, or call the Cancer Information Service at 1-800-4-CANCER (1-800-422-6237).

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