Proton radiotherapy for pediatric central nervous system ependymoma: clinical outcomes for 70 patients

Ependymoma is treated with maximal surgical resection and localized radiotherapy. Minimizing unnecessary exposure to radiation is of paramount importance for young children. Proton radiotherapy (PRT) spares healthy tissues outside the target region, but reports of clinical outcomes are scarce. We report outcomes for 70 patients treated with PRT for intracranial ependymoma. Seventy patients with localized ependymoma treated with involved-field PRT at the Massachusetts General Hospital between October 2000 and February 2011 were included. Median age at diagnosis was 38 months (range, 3 mo20 y). Nineteen (27) patients had supratentorial ependymoma and 51(73) had infratentorial ependymoma. Forty-six (66) had gross total resection (GTR), and 24 (34) had subtotal resection (STR). At a median follow-up of 46 months, 3-year local control, progression-free survival, and overall survival were 83, 76, and 95, respectively. STR was significantly associated with worse progression-free survival (54 vs 88, P .001) and overall survival (90 vs 97 for GTR, P .001). In a subset of patients (n 14), mean intelligence was 108.5 at baseline and 111.3 after mean 2.05 years of follow-up. In a larger group of patients (n 28), overall adaptive skills were 100.1 at baseline and 100.8 after 2.21 years of follow-up. Few patients developed evidence of growth hormone deficiency, hypothyroidism, or hearing loss. Outcomes for children treated with PRT compare favorably with the literature. STR correlated with inferior outcome. The young age at diagnosis and the proximity of critical structures in patients with ependymoma make PRT an ideal radiation modality.