Background: Current treatment for childhood intracranial ependymomas with surgery and radiation therapy (RT) yields 5-year survival rates ranging from 50-70% after complete resection to 0-30% after incomplete surgical resection. The role of chemotherapy in the treatment of ependymoma has not been established. In this pilot study, children with newly diagnosed intracranial ependymoma were treated with RT and chemotherapy using agents comparable to those found to be active in the treatment of intracranial ependymoma in infants.
Methods: Nineteen children age 3-14 years (median, 7.5 years) were treated with postoperative RT and chemotherapy. Chemotherapy was comprised of carboplatin, 560 mg/m2, with vincristine, 1.5 mg/m2, weekly for 3 weeks, alternating at 4-week intervals with ifosfamide, 1.8 g/m2, and etoposide, 100 mg/m2, for 5 consecutive days for a total of 4 cycles.
Results: The 5-year progression free survival (PFS) estimate was 74%. The extent of surgical resection was not a significant prognostic factor in this study. By contrast, ependymomas located in the posterior fossa were associated with a higher rate of progression (P = 0.036). Toxicity, limited predominantly to myelosuppression, was manageable.
Conclusions: The PFS for children with postoperative residual ependymoma treated with RT and chemotherapy in this study was higher than published survival results for RT alone. These results suggest a role for multialkylator chemotherapy in incompletely resected intracranial ependymoma and provide the rationale for a randomized trial comparing this strategy with conventional postoperative RT.