Stratum 2 patients were treated with pre-CRT chemotherapy consisting of two three-week cycles of vincristine, carboplatin and cyclophosphamide (cycle 1) and etoposide (cycle 2); and some of the stratum 2 patients had a second surgery prior to CRT. Stratum 3 and stratum 4 patients received post-operative CRT. Radiation was administered using a 1.0 cm clinical target volume margin. The cumulative total dose was 59.4 Gy, except for patients younger than 18 months after GTR.

One endpoint the study measured to evaluate the effectiveness of treatment was the rate of patients' five-year event free survival (EFS). In cancer, EFS refers to a length of time after primary treatment that the patient remains free of certain complications or events (such as the return of the cancer or onset of symptoms) that the treatment was intended to prevent or delay.


Data indicated that progression (meaning the cancer was spreading or getting worse) was observed in five of the 11 eligible stratum 1 patients, and the five-year EFS rate for patients in stratum 1 was 61.4 percent + 14.4 percent. In stratum 2, a second surgery was performed in 25 of the 64 patients, and GTR was achieved in 14 patients. There was no difference in EFS comparing the 25 patients that underwent a second surgery to the 39 patients that did not (log-rank test: P=0.0790). The EFS rate for stratum 2 patients was 39.2 percent + 7.0 percent. The EFS rate for patients in stratum 3 was 67.3 percent + 4.5 percent; and 69.5 percent + 3.8 percent for patients in stratum 4. Among the 281 patients treated on stratum 3 and 4, EFS was 74.6 + 3.6 percent for those with WHO grade II tumors and 60.7 percent + 4.7 percent for those with WHO grade III tumors, according to central pathology review (log-rank test: P=0.0047).

"These results indicate that radiation therapy may be safely administered to children of all ages with ependymoma and high-rate of tumor control may be achieved for the majority of children," said Thomas E. Merchant, DO, PhD, lead author of the study and Baddia J. Rashid Endowed Chair in Radiation Oncology at St. Jude Children's Research Hospital. "All children with ependymoma should receive expert care and treatment teams should follow protocol guidelines similar to those used in this study with consideration given to the importance of gross-total tumor resection and advances in radiation therapy methods. Other treatments, in addition to surgery and radiation therapy, should be investigated to further increase the rate of tumor control."

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