Peer-reviewed article
EPEN-54. ACNS0831, PHASE III RANDOMIZED TRIAL OF POST-RADIATION CHEMOTHERAPY IN PATIENTS WITH NEWLY DIAGNOSED EPENDYMOMA AGES 1 TO 21 YEARS
Smith A, Onar-Thomas A, Ellison D, Owens-Pickle E, Wu S et al. ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This is a phase III randomized clinical trial testing chemotherapy as an additional treatment for children with ependymoma (a brain tumor) after surgery and radiation. The study enrolled 451 eligible children aged 1-21 years and compared radiation therapy alone versus radiation therapy plus chemotherapy. Results showed a modest, statistically non-significant improvement in event-free survival with chemotherapy in the primary analysis, though a secondary analysis suggested potential benefit in patients who actually received the chemotherapy.
Key points
- 479 patients enrolled; 451 were eligible; 325 were randomized to treatment arms
- Primary intent-to-treat analysis: 3-year event-free survival was 78% (RT + chemotherapy) vs. 72% (RT alone), not statistically significant (p=0.074)
- Secondary 'as treated' analysis showed 3-year event-free survival of 80% vs. 71% (p=0.0121) in favor of chemotherapy
- High non-compliance in the chemotherapy arm (30.5%) compared to RT-only arm (4.3%)
- Genomic analyses ongoing to identify which patients may benefit from maintenance chemotherapy
Limitations
This report presents early results from an ongoing trial with significant treatment non-compliance that complicates interpretation of the primary analysis. The document does not provide details on neurobehavioral, quality of life outcomes, or the completed biomarker analyses mentioned as secondary objectives.
Abstract
Abstract PURPOSE The primary objective of this study is to determine the EFS and OS of children with gross total and near totally resected ependymoma (EPN) treated with post-operative focal radiation therapy (RT) followed by randomization to either RT alone or RT + 4 cycles of maintenance chemotherapy with vincristine, cisplatin, cyclophosphamide and etoposide. Secondary objectives include estimating the EFS and OS of children not randomized, evaluation of neurobehavioral and quality of life (QoL) endpoints, and EPN biomarkers. RESULTS 479 patients enrolled, 451 were eligible. Of 325 eligible randomized patients, 161 were randomized to RT alone and 164 to RT + maintenance chemotherapy. Age range (1–21 years, median 4.9 years). The planned primary analysis was based on intent-to-treat, irrespective of actual treatment received. Based on the data available as of 12/31/2019, estimated 3-year EFS in the RT + maintenance chemotherapy vs. RT arms were 78% vs. 72%, respectively, which did not meet statistical criteria to establish the benefit of maintenance chemotherapy post RT (1-sided log-rank p-value=0.074). Due to significant noncompliance (30.5% in the RT + maintenance vs 4.3% in the RT arm), a planned secondary “as treated” analysis was performed. With median follow-up of 42.6 months among patients without events, the 3 year EFS estimates for patients who received any chemotherapy (n=114) vs. those who received RT only (n=196) were 80% vs. 71%, respectively (1-sided p-value = 0.0121). CONCLUSION Early results in this randomized trial suggest that there may be some EPN patients who benefit from maintenance chemotherapy. Genomic analyses are ongoing.