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

Diffuse Intrinsic Pontine Gliomas

Conventional treatment for children with diffuse intrinsic pontine glioma (DIPG) is radiation therapy to involved areas. Such treatment will result in transient benefit for most patients, but over 90% of patients will die within 18 months of diagnosis. The conventional dose of radiation therapy ranges between 54 Gy and 60 Gy given locally to the primary tumor site in single daily fractions.

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Hyperfractionated (twice daily) radiation therapy techniques have been used to deliver a higher dose, and studies using doses as high as 78 Gy have been completed. Evidence demonstrates that these increased radiation therapy doses do not improve the duration or rate of survival for patients with DIPG whether given alone,[1,2] or in combination with chemotherapy.[3] Hypofractionated radiation therapy does not improve survival.[4][Level of evidence: 2A] Studies evaluating the efficacy of various radiosensitizers as a means for enhancing the therapeutic effect of this modality have been undertaken but to date have failed to show any significant improvement in outcome.[2,3,5,6,7] Radiation-induced changes may occur a few months after the completion of radiation therapy and may mimic tumor progression. When considering the efficacy of additional treatment, care needs to be taken to separate radiation-induced change from progressive disease.[8]

The utility of chemotherapy in the treatment of patients with newly diagnosed DIPG is unproven.[2,3,6,7,9,10,11]; [12,13][Level of evidence: 2A]; [14][Level of evidence: 3iiiA] Currently, no chemotherapeutic strategy—including neoadjuvant, concurrent, post-radiation survival or immunotherapy—when added to radiation therapy has led to long-term survival for children with DIPG.[15,16,17]; [18][Level of evidence: 2A] Similarly, studies utilizing high-dose, marrow-ablative chemotherapy with autologous hematopoietic stem cell rescue have been ineffective in extending survival.[19] Consonant with other brain tumors, radiation therapy is often omitted for infants with DIPG and chemotherapy-only approaches are utilized.

Focal or Low-Grade Brain Stem Gliomas

In general, maximal surgical resection should be attempted.[20,21] Patients with residual tumors may be candidates for additional therapy including 3-dimensional conformal radiation therapy approaches with or without adjuvant chemotherapy. Information about ongoing clinical trials is available from the NCI Web site.

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