Table 2.
Published pediatric ependymoma patient cohorts enrolled in clinical trials (review of the literature)
| Study | No. of patients (n) | EFS/OS | Prognostic factors | Implications | |
|---|---|---|---|---|---|
| Grill et al. (2001) | Multi-center | n=73 (<5 years) | 4-year EFS/OS 22%/59% |
OS: EOR, St tumors |
Chemotherapy only or to delay radiation therapy may be suitable for a subset of tumors; however, results were not competitive when compared to other studies |
| Timmermann et al. (2005) | Multi-center | n=34 (<3 years) | 3-year EFS/OS 27.3%/55.9% |
EFS: EOR |
Radiation therapy (in individual cases including the neuroaxis) should be administered |
| Merchant et al. (2009) | Single center | n=153 | 5-year EFS/OS 74%/85.0% |
EFS: gender, age, EOR OS: EOR, WHO grade, ethnic group |
Maximal safe surgery and high dose (54–59.4Gy) at an early age (12 months) achieved good results with low risk for 2nd malignancies and brainstem necrosis; age and sex may be used as risk stratification in future trials |
| Upadhyaya et al. (2019) | Multi-center | n=54 (<3 years) | 4-year EFS/OS 75.1%/92.6% |
EFS: EOR (incl. re-resection), PFA + chromosome 1q gain OS: - |
Radiation therapy may be feasible in young children (54Gy) |
| Merchant et al. (2019) | Multi-center | n=356 | 5-year EFS/OS 62.7%/83.8% |
EFS: EOR, chromosome 1q gain in PF-EPN, WHO grade, Gender |
Some supratentorial tumors may be cured by complete resection and observation alone, early postoperative radiation therapy (54–59.4Gy) is beneficial, also for patients younger than 3 years |
| Massimino et al. (2016 + 2020) | Multi-center | n=160 |
5-year EFS/OS 65.4%/81.1% 10-year EFS/OS 58%/73% |
EFS: EOR (incl. re-resection), WHO grade, gender; PF-EPN-A, CDKN2A deletion, chromosome 1q gain OS: EOR, WHO grade, VP-shunt, gender, age, PF-EPN-A, CDKN2A deletion, chromosome 1q gain chromosome 1q gain and CDKN2A deletion may be more frequent in children >3 years and are associated with a higher risk of dissemination |
Re-resection is warranted, in case of residual tumor a boost of 8Gy (additional to 59.4Gy) may be beneficial and feasible, future trials should include molecular classifications for risk stratification |