After surgery, chemotherapy was given as follows: regimen I with four blocks of vincristine, high-dose methotrexate 5 g/m2, and cyclophosphamide 1.5 g/m2 alternating with cisplatin 90 mg/m2 plus VP16 450 mg/m2 for 14 months; subsequently, regimen II was used: VEC (VCR, VP16 300 mg/m2, and cyclophosphamide 3 g/m2) for 6 months. Radiotherapy was planned for residual tumor after the completion of chemotherapy or for progression.
We treated 23 boys and 18 girls who were a median 22 months old; 14 were given regimen I, 27 were given regimen II; 22 underwent complete resection, 19 had residual tumor. Ependymoma was Grade 2 in 25 patients and Grade 3 in 16; tumors were infratentorial in 37 patients and supratentorial in 4. One child had intracranial metastases; 29 had progressed locally after a median 9 months. Event-free survival was 26 % at 3 and 5 years and 23 % at 8 years. One child died of sepsis, and another developed a glioblastoma 72 months after RT. Progression-free survival was 27 % at 3, 5, and 8 years, and overall survival was 48 % , 37 % , and 28 % at 3, 5, and 8 years, respectively. Of the 13 survivors, 6 never received RT; their intellectual outcome did not differ significantly in those children than in those without RT.
Our results confirm poor rates of event-free survival and overall survival for up-front chemotherapy in infant ependymoma. No better neurocognitive outcome was demonstrated in the few survivors who never received RT.