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Treatment for children with brain and spinal cord tumors is based on histology and location within the brain. For most of these tumors, an optimal regimen has not been determined, and enrollment onto clinical trials is encouraged. Get detailed information about these tumors in this clinician summary.

This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of childhood brain and spinal cord tumors. It is intended as a resource to inform and assist clinicians who care for cancer patients. It does not provide formal guidelines or recommendations for making health care decisions.

This summary is reviewed regularly and updated as necessary by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).

Childhood Brain & Spinal Cord Tumors Treatment

Childhood Brain and Spinal Cord Tumors Treatment Overview

childhood brain tumor

General Information About Childhood Brain and Spinal Cord Tumors

Primary brain tumors are a diverse group of diseases that together constitute the most common solid tumor of childhood. The Central Brain Tumor Registry of the United States (CBTRUS) estimates that approximately 4,300 U.S. children are diagnosed each year.

Brain tumors are classified by histology, but tumor location and extent of spread are also important factors that affect treatment and prognosis. Immunohistochemical analysis, cytogenetic and molecular genetic findings, and measures of proliferative activity are increasingly used in tumor diagnosis and classification.

Dramatic improvements in survival have been achieved for children and adolescents with cancer. Between 1975 and 2010, childhood cancer mortality decreased by more than 50%. Childhood and adolescent cancer survivors require close monitoring because cancer therapy side effects may persist or develop months or years after treatment. Refer to the PDQ summary on 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.

References

  1. Ostrom QT, Gittleman H, Farah P, et al.: CBTRUS statistical report: Primary brain and central nervous system tumors diagnosed in the United States in 2006-2010. Neuro Oncol 15 (Suppl 2): ii1-56, 2013.
  2. Louis DN, Perry A, Reifenberger G, et al.: The 2016 World Health Organization Classification of Tumors of the Central Nervous System: a summary. Acta Neuropathol 131 (6): 803-20, 2016.
  3. Smith MA, Altekruse SF, Adamson PC, et al.: Declining childhood and adolescent cancer mortality. Cancer 120 (16): 2497-506, 2014.

General Approach to Care for Children With Brain and Spinal Cord Tumors

Important concepts that should be understood by those treating and caring for a child who has a brain tumor or spinal cord tumor include the following:

  1. The cause of most childhood brain tumors remains unknown; however, germline mutations are becoming increasingly recognized as cancer-predisposing, as they are identified in up to 8% of children with cancer.
  2. Selection of an appropriate therapy can only occur if the correct diagnosis is made and the stage of the disease is accurately determined.
  3. Children with primary brain or spinal cord tumors represent a major therapy challenge that, for optimal results, requires the coordinated efforts of pediatric specialists in fields such as neurosurgery, neuropathology, radiation oncology, pediatric oncology, neuro-oncology, neurology, rehabilitation, neuroradiology, endocrinology, and psychology, who have special expertise in the care of patients with these diseases. For example, radiation therapy of pediatric brain tumors is technically demanding and should be performed in centers that have experience in this area.
  4. For most childhood brain and spinal cord tumors, the optimal treatment regimen has not been determined. Children who have brain and spinal cord tumors should be considered for enrollment in a clinical trial when an appropriate study is available. Such clinical trials are carried out by institutions and cooperative groups. Survival of childhood cancer has increased as a result of clinical trials that have attempted to improve upon the best accepted therapy available. Clinical trials in pediatrics are designed to compare new therapies with treatments that are 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 then comparing the results with those previously obtained using existing therapy. Information about ongoing clinical trials is available from the NCI website.
  5. While more than 70% of children diagnosed with brain tumors will survive for more than 5 years after diagnosis, survival rates are wide-ranging depending on tumor type and stage. Long-term sequelae related both to the effects of the tumor and its treatment are common. Debilitating effects on growth and neurologic development have frequently been observed after radiation therapy, especially in younger children. Secondary tumors have increasingly been diagnosed in long-term survivors. The dose and volume of radiation therapy appropriate for specific tumor types continues to be refined, and techniques for its administration (e.g., more conformal targeted-field design and protons) have evolved to mitigate the potential for adverse effects. In addition, the role of chemotherapy in allowing a delay or reduction in the administration of radiation therapy is under study, and preliminary results suggest that chemotherapy can be used to delay, limit, and sometimes obviate, the need for radiation therapy in children with benign and malignant lesions. Long-term management of these patients is complex and requires a multidisciplinary approach.

    (Refer to the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information about possible long-term or late effects.)

  6. Guidelines for pediatric cancer centers and their role in the treatment of pediatric patients with cancer have been outlined by the American Academy of Pediatrics.

References

  1. Fisher JL, Schwartzbaum JA, Wrensch M, et al.: Epidemiology of brain tumors. Neurol Clin 25 (4): 867-90, vii, 2007.
  2. Zhang J, Walsh MF, Wu G, et al.: Germline Mutations in Predisposition Genes in Pediatric Cancer. N Engl J Med 373 (24): 2336-46, 2015.
  3. Parsons DW, Pollack IF, Hass-Kogan DA, et al.: Gliomas, ependymomas, and other nonembryonal tumors of the central nervous system. In: Pizzo PA, Poplack DG, eds.: Principles and Practice of Pediatric Oncology. 7th ed. Philadelphia, Pa: Lippincott Williams and Wilkins, 2015, pp 628-70.
  4. Pollack IF: Brain tumors in children. N Engl J Med 331 (22): 1500-7, 1994.
  5. Smith MA, Seibel NL, Altekruse SF, et al.: Outcomes for children and adolescents with cancer: challenges for the twenty-first century. J Clin Oncol 28 (15): 2625-34, 2010.
  6. Reimers TS, Mortensen EL, Nysom K, et al.: Health-related quality of life in long-term survivors of childhood brain tumors. Pediatr Blood Cancer 53 (6): 1086-91, 2009.
  7. Iuvone L, Peruzzi L, Colosimo C, et al.: Pretreatment neuropsychological deficits in children with brain tumors. Neuro Oncol 13 (5): 517-24, 2011.
  8. Armstrong GT: Long-term survivors of childhood central nervous system malignancies: the experience of the Childhood Cancer Survivor Study. Eur J Paediatr Neurol 14 (4): 298-303, 2010.
  9. 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.
  10. Packer RJ, Lange B, Ater J, et al.: Carboplatin and vincristine for recurrent and newly diagnosed low-grade gliomas of childhood. J Clin Oncol 11 (5): 850-6, 1993.
  11. Mason WP, Grovas A, Halpern S, et al.: Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumors. J Clin Oncol 16 (1): 210-21, 1998.
  12. Corrigan JJ, Feig SA; American Academy of Pediatrics: Guidelines for pediatric cancer centers. Pediatrics 113 (6): 1833-5, 2004.

Staging, Classification, and Treatment of Newly Diagnosed and Recurrent Childhood Brain and Spinal Cord Tumors

Presently, there is no uniformly accepted staging system for most childhood brain tumors.

The classification of childhood central nervous system (CNS) tumors is based on histology, location, and extent of spread (refer to the Table below). Immunohistochemical analysis, cytogenetic and molecular genetic findings, and measures of proliferative activity are increasingly used in tumor diagnosis and classification. With advances in molecular data, it is conceivable that genomic factors will refine classification approaches and will be increasingly used to stratify patients entered on clinical trials.

Primary CNS spinal cord tumors comprise approximately 1% to 2% of all childhood CNS tumors. The classification of spinal cord tumors is based on histopathologic characteristics of the tumor and does not differ from that of primary brain tumors.

CNS Tumor Type, Pathologic Subtype, and Its Related PDQ Treatment Summary

Tumor Type (Based on the 2016 WHO Classification)Pathologic Subtype (Based on the 2016 WHO Classification)Related PDQ Treatment SummaryCNS = central nervous system; WHO = World Health Organization.Diffuse astrocytic tumorsDiffuse astrocytoma, IDH-mutant and IDH-wildtypeChildhood Astrocytomas TreatmentAnaplastic astrocytoma, IDH-mutant and IDH-wildtypeGlioblastoma, IDH-mutant and IDH-wildtypeDiffuse midline glioma, H3K27M-mutantChildhood Astrocytomas Treatment Childhood Brain Stem Glioma TreatmentOther astrocytic tumorsPilocytic astrocytomaChildhood Astrocytomas TreatmentChildhood Brain Stem Glioma TreatmentSubependymal giant cell astrocytomaChildhood Astrocytomas TreatmentPleomorphic xanthoastrocytomaAnaplastic pleomorphic xanthoastrocytomaEpendymal tumorsSubependymomaChildhood Ependymoma TreatmentMyxopapillary ependymomaEpendymomaEpendymoma, RELA fusion–positiveAnaplastic ependymomaOther gliomasAngiocentric gliomaChildhood Astrocytomas TreatmentAstroblastomaNeuronal and mixed neuronal-glial tumorsDysembryoplastic neuroepithelial tumorChildhood Astrocytomas TreatmentGangliogliomaDesmoplastic infantile astrocytoma and gangliogliomaPapillary glioneuronal tumorRosette-forming glioneuronal tumorDiffuse leptomeningeal glioneuronal tumorExtraventricular neurocytomaCerebellar liponeurocytomaParagangliomaTumors of the pineal regionPineoblastomaChildhood Medulloblastoma and Other Central Nervous System Embryonal Tumors TreatmentEmbryonal tumorsMedulloblastoma, WNT-activatedChildhood Medulloblastoma and Other Central Nervous System Embryonal Tumors TreatmentMedulloblastoma, SHH-activated and TP53-mutant; Medulloblastoma, SHH-activated and TP53-wildtypeMedulloblastoma, non-WNT/non-SHH group 3Medulloblastoma, non-WNT/non-SHH group 4Medulloblastoma, classicMedulloblastoma, desmoplastic/nodularMedulloblastoma with extensive nodularityMedulloblastoma, large cell/anaplasticEmbryonal tumor with multilayered rosettes, C19MC-alteredMedulloepitheliomaCNS neuroblastomaCNS ganglioneuroblastomaAtypical teratoid/rhabdoid tumorChildhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor TreatmentCNS embryonal tumor with rhabdoid featuresChildhood Medulloblastoma and Other Central Nervous System Embryonal Tumors TreatmentGerm cell tumorsGerminomaChildhood Central Nervous System Germ Cell Tumors TreatmentEmbryonal carcinomaYolk sac tumorChoriocarcinomaMature teratomaImmature teratomaTeratoma with malignant transformationMixed germ cell tumorTumors of the sellar regionAdamantinomatous craniopharyngiomaChildhood Craniopharyngioma TreatmentPapillary craniopharyngioma

Relapse is not uncommon in both low-grade and malignant childhood brain tumors and may occur many years after initial treatment. Disease relapse may occur at the primary tumor site or, especially in malignant tumors, at noncontiguous CNS sites. Systemic relapse is rare but may occur in some tumor types. At recurrence, a complete evaluation for extent of relapse is indicated for all malignant tumors and, at times, for lower-grade lesions. Biopsy or surgical re-resection may be necessary for confirmation of relapse or the diagnosis of tumor transformation, which can include a change in grade and molecular makeup. Other entities, such as secondary tumor and treatment-related intratumoral necrosis or frank brain necrosis, may be clinically indistinguishable from tumor recurrence. Determining the need for surgical intervention must be individualized on the basis of the initial tumor type, the length of time between initial treatment and the reappearance of the lesion, and other clinical parameters.

Early-phase therapeutic trials may be available for selected patients via Children's Oncology Group phase I institutions, the Pediatric Brain Tumor Consortium, or other entities.

References

  1. Louis DN, Perry A, Reifenberger G, et al.: The 2016 World Health Organization Classification of Tumors of the Central Nervous System: a summary. Acta Neuropathol 131 (6): 803-20, 2016.
  2. Morrissy AS, Garzia L, Shih DJ, et al.: Divergent clonal selection dominates medulloblastoma at recurrence. Nature 529 (7586): 351-7, 2016.
  3. Mistry M, Zhukova N, Merico D, et al.: BRAF mutation and CDKN2A deletion define a clinically distinct subgroup of childhood secondary high-grade glioma. J Clin Oncol 33 (9): 1015-22, 2015.
  4. Packer RJ, Zhou T, Holmes E, et al.: Survival and secondary tumors in children with medulloblastoma receiving radiotherapy and adjuvant chemotherapy: results of Children's Oncology Group trial A9961. Neuro Oncol 15 (1): 97-103, 2013.

Changes to This Summary (10/09/2020)

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.

This summary was reformatted.

This summary is written and maintained by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of NCI. The summary reflects an independent review of the literature and does not represent a policy statement of NCI or NIH. More information about summary policies and the role of the PDQ Editorial Boards in maintaining the PDQ summaries can be found on the About This PDQ Summary and PDQ® - NCI's Comprehensive Cancer Database pages.

About This PDQ Summary

Purpose of This Summary

This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of childhood brain and spinal cord tumors. It is intended as a resource to inform and assist clinicians who care for cancer patients. It does not provide formal guidelines or recommendations for making health care decisions.

Reviewers and Updates

This summary is reviewed regularly and updated as necessary by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).

Board members review recently published articles each month to determine whether an article should:

  • be discussed at a meeting,
  • be cited with text, or
  • replace or update an existing article that is already cited.

Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary.

The lead reviewers for Childhood Brain and Spinal Cord Tumors Treatment Overview are:

  • Kenneth J. Cohen, MD, MBA (Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Hospital)
  • Louis S. Constine, MD (James P. Wilmot Cancer Center at University of Rochester Medical Center)
  • Roger J. Packer, MD (Children's National Health System)
  • D. Williams Parsons, MD, PhD
  • Malcolm A. Smith, MD, PhD (National Cancer Institute)

Any comments or questions about the summary content should be submitted to Cancer.gov through the NCI website's Email Us. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries.

Levels of Evidence

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 Treatment Editorial Board uses a formal evidence ranking system in developing its level-of-evidence designations.

Permission to Use This Summary

PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary].”

The preferred citation for this PDQ summary is:

PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain and Spinal Cord Tumors Treatment Overview. Bethesda, MD: National Cancer Institute. Updated . Available at: https://www.cancer.gov/types/brain/hp/child-brain-treatment-pdq. Accessed . [PMID: 26389453]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in Visuals Online, a collection of over 2,000 scientific images.

Disclaimer

Based on the strength of the available evidence, treatment options may be described as either “standard” or “under clinical evaluation.” These classifications should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

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