Outcome of 11 children with ependymoblastoma treated within the prospective HIT-trials between 1991 and 2006
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Nicolas U Gerber1, Katja von Hoff, André O von Bueren
1Department of Oncology, University Children's Hospital, 8032 Zurich, Switzerland. nicolas.gerber@kispi.uzh.ch
Insights
Ependymoblastoma, a rare childhood brain tumor, has a poor prognosis. Multimodal treatment offers sustained remissions, highlighting the need for central pathology review and further research for improved survival in CNS ependymoblastoma.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Cancer Research
Background:
- Ependymoblastoma is a rare malignant brain tumor predominantly affecting young children.
- Limited data exists on the clinical behavior and optimal treatment strategies for this aggressive CNS tumor.
Purpose of the Study:
- To report clinical outcomes and treatment strategies for children with ependymoblastoma.
- To emphasize the importance of central pathology review in diagnosing CNS ependymoblastoma.
Main Methods:
- Retrospective analysis of 11 children with centrally confirmed ependymoblastoma treated between 1994-2006.
- Multimodal treatment regimens including surgery, chemotherapy, radiotherapy, and high-dose chemotherapy were employed.
Main Results:
- The 5-year progression-free survival was 36.4% and overall survival was 30.3%.
- Gross-total resection, craniospinal radiotherapy, and high-dose chemotherapy were associated with better outcomes in survivors.
- Significant diagnostic discrepancies were noted between local and central pathology reviews.
Conclusions:
- Prognosis for ependymoblastoma remains poor, but multimodal therapy can achieve sustained remissions.
- Central pathology review is crucial for accurate diagnosis of CNS ependymoblastoma.
- Further research is essential to improve survival rates for children with this rare brain tumor.
Abstract:
Ependymoblastoma is a rare malignant brain tumor of early childhood. Data on clinical behavior and optimal treatment strategies are scarce. We report on 11 consecutively treated children with centrally confirmed diagnosis of CNS ependymoblastoma, registered between February 1994 and October 2006 to the prospective GPOH-HIT multicenter brain tumor trials, and treated by multimodal regimens. Median age at diagnosis was 3.5 years (range, 1.8-5.6 years), and the median follow-up of survivors was 5.9 years (range, 2.2-12.7 years). Initial stage was M0 in 9, and M0/1 (no cerebrospinal fluid examination done) in 2 patients. Gross-total tumor resection was achieved in 7 patients, incomplete resection in 4 patients. Further primary therapy included chemotherapy in all patients, craniospinal radiotherapy in 5 patients and high-dose chemotherapy in 2 patients. Tumor response to chemotherapy was observed in 1 of 4 evaluable patients. Tumor progression occurred in 7 patients after a median time of 5.0 months (range, 2.5-19.2 months). Five-year progression-free survival was 36.4% (±14.5%), 5-year overall survival 30.3% (±15.9%). Of 4 survivors, 3 had gross-total tumor resection, and all were treated by either craniospinal radiotherapy and/or high-dose chemotherapy with autologous blood stem cell rescue. Prognosis of children with ependymoblastoma is poor, but sustained remissions have been achieved after multimodal treatment. Considerable diagnostic discrepancies between local and central pathologists underscore the importance of central review. Further studies are needed to improve survival of children with this rare malignant central nervous system tumor.
