National Cancer Institute
Last Modified: April 9, 2008
This PDQ® cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of childhood supratentorial primitive neuroectodermal tumors and pineoblastoma. This summary is reviewed regularly and updated as necessary by the PDQ® Pediatric Treatment Editorial Board.
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.
In the summary, treatments are described as standard or conventional and under clinical evaluation. These designations 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.]
This PDQ® summary contains content that is also included in the new PDQ® Childhood Central Nervous System Embryonal Tumors summary. In the very near future, the PDQ® Childhood Supratentorial Primitive Neuroectodermal Tumors and Pineoblastoma summary will be removed from the National Cancer Institute (NCI) website and the content contained in this summary will be found in the PDQ® Childhood Central Nervous System Embryonal Tumors summary.
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.
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® Childhood Brain and Spinal Cord Tumors Treatment summary for information about the general classification of childhood brain and spinal cord tumors.
The classification of brain tumors is based on both histopathologic characteristics and location in the brain. Undifferentiated neuroectodermal tumors of the cerebellum have historically been referred to as medulloblastomas, while tumors of identical histology in the pineal region are diagnosed as pineoblastomas, and cortical lesions have been called central neuroblastomas or cortical primitive neuroectodermal tumors. Studies have suggested that the tumor cells of medulloblastomas and supratentorial primitive neuroectodermal tumors have different molecular genetic aberrations. 1 2 This group also includes some tumors formerly called central neuroblastomas. The nomenclature of pediatric brain tumors is controversial and potentially confusing. Some pathologists advocate abandoning the traditional morphologically based classifications such as medulloblastoma in favor of a terminology that relies more extensively on the phenotypic characteristics of the tumor. In such a system, medulloblastoma is referred to as primitive neuroectodermal tumor and then subdivided on the basis of cellular differentiation. The World Health Organization 2000 classification of brain tumors maintains the term medulloblastoma for posterior fossa undifferentiated tumors. 3 It also maintains separate categories for cerebral primitive neuroectodermal tumors and for pineal small round cell tumors (pineoblastomas). The pathologic classification of pediatric brain tumors is a specialized area that is evolving; review of the diagnostic tissue by a neuropathologist who has particular expertise in this area is strongly recommended.
Poorly differentiated small round cell tumors of the cerebrum have been referred to as cerebral neuroblastoma and as primitive neuroectodermal tumors. These tumors in the pineal body have classically been called pineoblastomas. Histologically, these tumors may be similar to cerebellar medulloblastoma with varying proportions of features that suggest astrocytic or ependymal differentiation. Genetic aberrations of supratentorial brain tumors appear to be different from those of infratentorial primitive neuroectodermal tumors (PNET, medulloblastoma). 1 Tumors may spread throughout the subarachnoid space. Every patient with a primitive neuroectodermal tumor or with pineoblastoma should be evaluated with diagnostic imaging of the spinal cord and whole brain. The most sensitive method currently available for evaluating spinal cord subarachnoid metastasis is spinal magnetic resonance imaging performed with gadolinium. Cerebrospinal fluid should be examined cytologically for tumor cells. There is no generally accepted staging system for supratentorial primitive neuroectodermal tumors and for pineoblastomas, but the staging system used for medulloblastoma is conventionally used for tumor dissemination. Prognosis is probably related to the extent of disease both at diagnosis and after surgery. 2 3 4 5 Patients with cystic tumors may fare better. [Note: 2-year survival rate: 30% to 50%]
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 that were 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 therapy, treatment planning by a multidisciplinary team of cancer specialists who have experience treating childhood brain tumors is required. Radiation therapy, if needed, is technically very demanding and should be carried out in centers that have experience in that area to ensure optimal results.
In the past, treatment has included surgery with radiation therapy. Evidence suggests that more extensive surgical resections are related to an improved rate of survival for patients with medulloblastoma without dissemination at diagnosis, primarily in children with nondisseminated posterior fossa disease at diagnosis. This has not yet been proven for childhood supratentorial primitive neuroectodermal and pineal (pineoblastoma) tumors. The results of prospective, randomized trials and large single-arm trials have suggested that adjuvant chemotherapy given during and after radiation therapy may improve overall survival in children with medulloblastoma. Several nonrandomized trials suggest potential benefit of postradiation chemotherapy. 1 2 3 4 5 Children younger than 3 years are particularly susceptible to the adverse effects of radiation on brain development. Debilitating effects on growth and neurologic development have frequently been observed, especially in younger children. 6 7 8 For this reason, the role of chemotherapy in allowing for a delay in the administration of radiation therapy is under study, and preliminary results suggest that chemotherapy can be used to delay, if not obviate, the need for radiation therapy in some children younger than 3 years with primitive neuroectodermal tumors. 9 Surveillance testing is presently a part of all ongoing supratentorial primitive neuroectodermal studies. 10 Long-term management of these patients is complex and requires a multidisciplinary approach.
The designations in PDQ® that treatments are standard or under clinical evaluation are not to be used as a basis for reimbursement determinations.
Careful evaluation to fully determine the extent of disease must precede the treatment of childhood supratentorial primitive neuroectodermal tumors (SPNET) and pineoblastoma. Surgery should be an attempt at maximal tumor reduction. Postoperatively, studies should be conducted to determine if the patient has disseminated disease. 1 Risk criteria are outlined in the stage information section. Patients with extensive tumors should be considered at the highest risk of relapse and should be treated on protocols specifically designed for them. 2 Children with pineal primitive neuroectodermal tumors may have a more favorable prognosis when treated with surgery, radiation therapy, and chemotherapy than children with SPNETs. 2 3
The usual postsurgical treatment is radiation therapy. 4 The suggested tumor dose is 54 Gy to 56 Gy using conventional fractionation. Craniospinal irradiation with 23.4 Gy to 36 Gy is also recommended because of the propensity of this tumor to disseminate through the subarachnoid space. Given the overall poorer prognosis for patients with extensive disease, the addition of chemotherapy before or after radiation therapy is being explored. 5 6 The role of chemotherapy to delay irradiation and its consequences is under clinical evaluation; preliminary results suggest that chemotherapy can be used to delay, modify, or, in selected cases, obviate the need for radiation therapy. 1
Children younger than 3 years:
Some patients younger than 3 years with newly diagnosed SPNETs and pineoblastomas will respond at least partially to chemotherapy. 1 2 3 7 Some patients, especially those with minimal residual postoperative disease, may have a long-lasting response. For this reason, studies that use chemotherapy to delay, modify, or possibly obviate the need for radiation therapy have been undertaken. Results of such studies for young children with SPNET and pineoblastomas have been disappointing. 1 8 Although chemotherapy is being used to prevent neurologic damage caused by radiation therapy in very young patients, neurologic deficits may be present in children prior to the initiation of therapy, and progressive neurologic damage has been noted during therapy. 9
Check for U.S. clinical trials from NCI's PDQ® Cancer Clinical Trials Registry that are now accepting patients with untreated childhood supratentorial primitive neuroectodermal tumor, childhood pineoblastoma and untreated childhood pineoblastoma. 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.
Recurrence is not uncommon in both benign and malignant childhood brain tumors and may develop many years after initial treatment. 1 Disease can be at the primary tumor site or, especially in malignant tumors, at noncontiguous central nervous system sites. Systemic relapse is rare but may occur. At time of relapse, a complete evaluation for extent of recurrence is indicated for all malignant tumors and, at times, for benign lesions. Biopsy or surgical resection may be necessary for confirmation of relapse because other entities, such as secondary tumor and treatment-related brain necrosis, may be clinically indistinguishable from tumor recurrence. 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 the clinical picture. Patients with supratentorial primitive neuroectodermal tumors or pineoblastomas that recur after radiation therapy alone should be considered for treatment with known active agents, which include vincristine, cyclophosphamide, cisplatin, carboplatin, and etoposide; response is seen in more than 50% of patients. 2 3 Entry into studies of novel therapeutic approaches at the time of relapse after radiation therapy alone or radiation therapy and chemotherapy should be considered. 4 5 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 supratentorial primitive neuroectodermal tumor, recurrent childhood pineoblastoma and childhood pineoblastoma. 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.
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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.
Editorial changes were made to this summary.
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).