Postoperative chemotherapy without irradiation for ependymoma in children under 5 years of age: A multicenter trial of the French Society of Pediatric Oncology

Purpose: To evaluate a strategy that avoids radiotherapy in first-line trea tment in children under 5 years of age with brain or posterior fossa ependy moma, by exclusively administering 16 months of adjuvant multiagent chemoth erapy after surgery. Patients and Methods: Between June 1990 and October 1998, 73 children with ependymoma (82% with high-grade tumors) were enrolled onto this multicenter trial. Children received adjuvant conventional chemotherapy after surgery consisting of seven cycles of three courses alternating two drugs at each c ourse (procarbazine and carboplatin, etoposide and cisplatin, vincristine a nd cyclophosphamide) over a year and a half. Systematic irradiation was not envisaged at the end of chemotherapy. In the event of relapse or progressi on, salvage treatment consisted of a second surgical procedure followed by local irradiation with or without second-line chemotherapy. Results: Conventional chemotherapy was well tolerated and could be administ ered in outpatient clinics. No radiologically documented response to chemot herapy more than 50% was observed. With a median follow-up of 4.7 years (ra nge, 5 months to 8 years), the 4-year progression-free survival rate in thi s series was 22% (95% confidence interval [CI], 13% to 43%) and the overall survival rate was 59% (95% CI, 47% to 71%). Overall, 40% (95% CI, 29% to 5 1%) of the patients were alive having never received radiotherapy 2 years a fter the initiation of chemotherapy and 23% (95% CI, 14% to 35%) were still alive at 4 years without recourse to this modality. In the multivariate an alysis, the two factors associated with a favorable outcome were a supraten torial tumor location (P = .0004) and complete surgery (P = .0009). Overall survival at 4 years was 74% (95% CI, 59% to 86%) for the patients in whom resection was radiologically complete and 35% (95% CI, 18% to 56%) for the patients with incomplete resection. Conclusion: A significant proportion of children with ependymoma can avoid radiotherapy with prolonged adjuvant chemotherapy. Deferring irradiation at the time of relapse did not compromise overall survival of the entire pati ent population. J Clin Oncol 19:1288-1296. (C) 2001 by American Society of Clinical Oncology.