Adjuvant Etoposide for Very High-risk PFA Ependymoma: A Case Report

J Pediatr Hematol Oncol. 2025 Aug 1;47(6):e269-e272. doi: 10.1097/MPH.0000000000003080. Epub 2025 Jul 1.

Abstract

Background: Ependymomas of the posterior fossa type A (PF-A) with a combined chromosome 1q gain and 6q loss are associated with an extremely high risk of recurrence and a very poor outcome.

Observations: We report the case of a 4-year-old girl who received adjuvant oral etoposide for 1 year after conventional treatment (surgery and focal radiation). The patient remains in clinical and radiologic remission 2.5 years post-diagnosis.

Conclusions: This approach of using oral etoposide could be considered in ultra-high-risk 6q loss PF-A ependymoma to try and decrease the risk of relapse, awaiting further evaluation in a clinical trial.

Keywords: 1q gain; 6q loss; ependymoma; etoposide; hydrocephaly; intracranial hypertension; proton therapy.

Publication types

  • Case Reports

MeSH terms

  • Antineoplastic Agents, Phytogenic* / therapeutic use
  • Chemotherapy, Adjuvant
  • Child, Preschool
  • Ependymoma* / drug therapy
  • Ependymoma* / genetics
  • Ependymoma* / pathology
  • Etoposide* / administration & dosage
  • Etoposide* / therapeutic use
  • Female
  • Humans
  • Infratentorial Neoplasms* / drug therapy
  • Infratentorial Neoplasms* / genetics
  • Infratentorial Neoplasms* / pathology

Substances

  • Etoposide
  • Antineoplastic Agents, Phytogenic