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    Childhood Brain Stem Glioma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Newly Diagnosed Childhood Brain Stem Glioma Treatment

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    Chemotherapy only (infants)

    Similar to the treatment of other brain tumors, radiation therapy is often omitted for infants with DIPGs, and chemotherapy-only approaches are utilized. However, published data supporting the utility of this approach is lacking.

    Treatment options under clinical evaluation

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

    Standard Treatment Options for Focal or Low-Grade Brain Stem Gliomas

    Standard treatment options for newly diagnosed focal or low-grade brain stem gliomas include the following:

    1. Surgical resection (with or without radiation therapy and chemotherapy).
    2. Observation (with or without cerebrospinal fluid diversion).
    3. Radiation therapy, chemotherapy, and alternative approaches for inoperable focal or low-grade tumors.

    Surgical resection (with or without radiation therapy and chemotherapy)

    In general, maximal surgical resection is attempted.[21,22]

    Patients with residual tumor may be candidates for additional therapy, including 3-dimensional conformal radiation therapy approaches, with or without adjuvant chemotherapy.

    Observation (with or without cerebrospinal fluid diversion)

    Patients with small tectal lesions and hydrocephalus but no other neurological deficits may be treated with cerebrospinal fluid diversion alone and have follow-up with sequential neuroradiographic studies unless there is evidence of progressive disease.[21]

    A period of observation may be indicated before instituting any treatment for patients with neurofibromatosis type 1.[23] Brain stem gliomas in these children may be indolent and may require no specific treatment for years.[24]

    Radiation therapy, chemotherapy, and alternative approaches for inoperable focal or low-grade tumors

    In selected circumstances, adjuvant therapy in the form of radiation therapy or chemotherapy can be considered in a child with a newly diagnosed focal or low-grade brain stem glioma.[25,26][Level of evidence: 3iDi] Decisions regarding the need for such therapy depend on the age of the child, the extent of resection obtainable, and associated neurologic deficits.

    Alternative approaches for the treatment of inoperable brain stem gliomas include the following:

    • Stereotactic iodine I-125 brachytherapy approaches, with or without adjuvant chemotherapy.[27]
    • The use of BRAF inhibitors for tumors harboring a V600E mutation.[28]
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