Grundy RG et al. (2007) Primary postoperative chemotherapy without radiotherapy for intracranial ependymoma in children: the UKCCSG/SIOP prospective study. Lancet Oncol 8: 696–705

Radiotherapy is an effective treatment for childhood intracranial ependymoma (IE), but its use in patients younger than 5 years of age is associated with damage to the central nervous system and an increased risk of a second malignancy. A study by Grundy et al. has examined the role of chemotherapy in children with IE.

The study enrolled 89 children with IE aged 3 years or younger at diagnosis who had not received prior drug or radiation treatment. Following surgery, the children received alternating cycles of myelosuppressive and nonmyelosuppressive chemotherapy for 1 year. Among the 80 patients without metastatic disease, 50 progressed; 34 received radiotherapy for this progression. The 5-year cumulative incidence rate of freedom from radiotherapy in those patients without metastatic disease was 42%. The 3-year and 5-year event-free survival rates for these patients were 47.6% and 41.8%, respectively. The overall 3-year and 5-year survival rates for nonmetastatic patients were 79.3% and 63.4%, respectively. Complete resection did not result in a better outcome, and overall and event-free survival did not differ according to age at diagnosis, site of disease or histological grade. The median time to progression was 1.6 years. The post-chemotherapy 5-year overall survival rates for the patients who received the highest (n = 23) and lowest (n = 32) relative dose intensity of chemotherapy were 76% and 52%, respectively.

This study shows that radiotherapy can be delayed or avoided in children with IE without compromising survival, when these patients are treated with chemotherapy.