Treatment and outcome of children with relapsed ependymoma: a multi-institutional retrospective analysis.
BACKGROUND
More than a third of children with ependymoma experience relapse, and despite multimodal treatment, less than 25% of them can then achieve long-term survival. Prognostic factors for patients who relapse have not been extensively analyzed.


METHODS
We retrospectively analyzed 82 patients from four pediatric oncology European institutions in order to identify prognostic factors and influence of treatment modalities in relapsed ependymoma.


RESULTS
First relapse occurred after a median of 19 months (1 month-16 years). Five-year progression-free survival and overall survival of the cohort were 17% and 27.6%, respectively. Survival was statistically significantly higher for patients achieving gross total resection. No survival benefit was seen for children receiving chemotherapy whereas patients who were amenable to some form of re-irradiation had a better outcome. Objective responses were found in more than 25% of patients receiving oral etoposide, temozolomide, or vincristine/etoposide/cyclophosphamide regimens. Multivariate analysis confirmed that patients with mixed relapses, no surgery at relapse, and receiving chemotherapy did worse (hazard ratio = 3.6, 3.3, and 1.7, respectively, all p < 0.05).


CONCLUSIONS
Relapsed ependymoma carries a very poor prognosis with an indolent chronic course, leading to death in approximately 90% of the patients. Complete surgical resection whenever possible should be encouraged. Radiation therapy of the relapsed lesions can provide some minor benefit whereas chemotherapy despite the occasional responses provides no benefit in the final outcome which is dismal. Efforts have to be orchestrated internationally to enroll these patients on clinical trials using biology-based therapies.
Zacharoulis
Stergios
S
Department of Paediatrics, The Royal Marsden Hospital, Sutton, Surrey, UK. stergios.zacharoulis@rmh.nhs.uk
Ashley
Sue
S
Moreno
Lucas
L
Gentet
Jean-Claude
JC
Massimino
Maura
M
Frappaz
Didier
D
D000293:Adolescent; D000704:Analysis of Variance; D000970:Antineoplastic Agents / Q000627:therapeutic use; D016543:Central Nervous System Neoplasms / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D003131:Combined Modality Therapy; D004806:Ependymoma / Q000628:therapy*; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D009364:Neoplasm Recurrence, Local; D019635:Neurosurgical Procedures; D011379:Prognosis; D016016:Proportional Hazards Models; D012189:Retrospective Studies; D016019:Survival Analysis; D016896:Treatment Outcome; D055815:Young Adult
D000970:Antineoplastic Agents