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 previously obtained with existing therapy.
Because of the relative rarity of cancer in children, all patients with aggressive brain tumors should be considered for entry into a clinical trial. To determine and implement optimum treatment, treatment planning by a multidisciplinary team of cancer specialists who have experience treating childhood brain tumors is required. Radiation therapy of pediatric brain tumors is technically very demanding and should be carried out in centers that have experience in that area in order to ensure optimal results.
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Treatment for childhood ependymoma has included surgery followed by standard fractionated radiation therapy. There is evidence to suggest that more extensive surgical resections are related to an improved rate of survival.[1,2,3,4,5,6,7] In addition, in a small series of children with localized ependymoma, adjuvant radiation therapy appeared to improve progression-free survival (PFS), even after adjusting for the extent of resection. In fact, a benefit in PFS was observed for patients who received adjuvant radiation therapy after gross total resection compared with those who did not receive radiation therapy. Additional research will be necessary to confirm these findings. Chemotherapy has been shown to be active in patients with recurrent ependymoma. One relatively small, prospective, randomized trial suggests that chemotherapy activity in newly diagnosed cases is limited, and current treatment approaches do not include chemotherapy as a component of primary therapy for most children with newly diagnosed ependymomas that are completely resected. Children younger than 3 years are particularly susceptible to the adverse effect of radiation on brain development.[Level of evidence: 3iiiC] Debilitating effects on growth and neurologic development have frequently been observed, especially in younger children.[12,13,14] For this reason, conformal radiation approaches, such as 3-dimensional conformal radiation therapy, that minimize damage to normal brain tissue and charged-particle radiation therapy, such as proton beam therapy, are under evaluation for infants and children with ependymoma.[15,16] Long-term management of these patients is complex and requires a multidisciplinary approach.