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Treatment Options for Medulloblastoma and CNS Primitive Neuroectodermal Tumor (PNET)

  • PEDIATRIC NEUROLOGY (HS SINGER, SECTION EDITOR)
  • Published:
Current Treatment Options in Neurology Aims and scope Submit manuscript

Opinion statement

Medulloblastoma and central nervous system (CNS) primitive neuroectodermal tumor (PNET) are primary pediatric brain tumors that require multidisciplinary therapies. Although often treated similarly in clinical trials, they are biologically different diseases. Even within medulloblastomas and CNS PNETs, there are molecularly distinct subgroups with differing presentations and prognoses. Overall, prognosis is better for medulloblastomas. Specific treatments for these types of cancer are continuously evolving to maximize survival and minimize long-term sequelae of treatment. Patients should be treated on a clinical trial, if eligible, as they may gain benefit with minimal risk over current standard of care. The amount of residual disease after surgery better correlates with survival for medulloblastomas than for CNS PNETs. Maximal surgical resection of tumor should be done, only if additional permanent, neurologic deficits can be spared. Patients should have a staging work-up to assess the extent of disease. This includes postoperative magnetic resonance imaging (MRI) of the brain, MRI of the entire spine and lumbar cerebrospinal fluid (CSF) sampling for cytological examination, if deemed safe. Radiation therapy to the entire CNS axis is required, with a greater dose (boost) given to the region of the primary site or any bulky residual disease for older children. Adjuvant chemotherapy must be given even if no evidence of disease after radiation therapy exists, as the risk of relapse is substantial after radiation alone. Subsets of younger children with medulloblastoma, arbitrarily defined as those younger than 3 years of age in some studies and 4 or even 5 years in other studies, can be effectively treated with chemotherapy alone. Recent genomic studies have revealed further subtypes of disease than previously recognized. Clinical trials to exploit these biologic differences are required to assess potential efficacy of targeted agents. The treatment of medulloblastoma and CNS PNET can cause significant impairment in neurologic function. Evaluations by physical therapy, occupational therapy, speech therapy and neurocognitive assessments should be obtained, as needed. After therapy is completed, survivors need follow-up of endocrine function, surveillance scans and psychosocial support.

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Conflicts of Interest

Kevin C. De Braganca and Roger J. Packer declare that they have no conflict of interest.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.

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Authors and Affiliations

  1. Memorial Sloan-Kettering Cancer Center, 1275 York Ave., New York, NY, 10065, USA

    Kevin C. De Braganca MD

  2. School of Medicine and Health Sciences, George Washington University, Washington, USA

    Roger J. Packer MD

  3. Children’s National Medical Center, Center for Neuroscience Research (CNR), 111 Michigan Avenue, NW, Washington, DC, 20010-2970, USA

    Roger J. Packer MD

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  1. Kevin C. De Braganca MD
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Correspondence to Kevin C. De Braganca MD.

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De Braganca, K.C., Packer, R.J. Treatment Options for Medulloblastoma and CNS Primitive Neuroectodermal Tumor (PNET). Curr Treat Options Neurol 15, 593–606 (2013). https://doi.org/10.1007/s11940-013-0255-4

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