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Adjuvant Etoposide for Very High-risk PFA Ependymoma: A Case Report
Jacob Silverman1, Melissa Mégalli2, Emilie Giguère2
1McGill Faculty of Medicine and Health Sciences.
Journal of Pediatric Hematology/Oncology
|July 1, 2025
Summary
Posterior fossa type A ependymoma with 6q loss is high-risk. Adjuvant oral etoposide showed promise in a pediatric case, maintaining remission for 2.5 years.
Area of Science:
- Neuro-oncology
- Pediatric oncology
- Molecular genetics
Background:
- Posterior fossa type A ependymomas (PF-A) with combined 1q gain and 6q loss present an ultra-high risk of recurrence.
- These genetic alterations are associated with a significantly poor prognosis in ependymoma patients.
Observation:
- A case study involving a 4-year-old girl diagnosed with PF-A ependymoma.
- The patient underwent conventional treatment including surgery and focal radiation therapy.
- Adjuvant therapy with oral etoposide was administered for one year post-conventional treatment.
Findings:
- The patient achieved and maintained clinical and radiologic remission for 2.5 years post-diagnosis.
- This suggests a potential benefit of oral etoposide in managing ultra-high-risk ependymoma.
Implications:
- Adjuvant oral etoposide may be a viable strategy to reduce relapse risk in pediatric PF-A ependymoma with 6q loss.
- Further clinical trials are warranted to validate this therapeutic approach in ependymoma treatment.

