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Ongoing Response in a Multiply Relapsed Metastatic Posterior Fossa Ependymoma A After Vorinostat and Concomitant
Hamza S Gorsi1,2, Stephanie A Toll1,2, Sandeep Sood3
1Division of Pediatric Hematology/Oncology, Children's Hospital of Michigan.
Abstract:
Posterior fossa ependymomas A confer the worst prognosis among all subtypes. They demonstrate distinct epigenetic changes, which can be targeted with epigenetic modifiers like histone deacetylase inhibitors (Vorinostat). We describe a 3-year-old male diagnosed with a posterior fossa ependymoma who had a number of recurrences requiring multimodal therapy. Molecular analysis demonstrated a BCL-6 corepressor mutation, and methylation profiling matched with posterior fossa ependymomas A. He received craniospinal irradiation and focal boost with Vorinostat. Serial imaging after irradiation revealed a progressively decreasing tumor burden with nearly complete resolution of disease at 15 months. Histone deacetylase inhibitors demonstrate promise in treatment of carefully selected cases of ependymoma.
Insights
Posterior fossa ependymomas (PFAs) are aggressive brain tumors. A novel treatment combining irradiation with Vorinostat, a histone deacetylase inhibitor, showed significant tumor reduction in a young patient with recurrent PFA.
Area of Science:
- Neuro-oncology
- Epigenetics
- Pediatric oncology
Background:
- Posterior fossa ependymomas (PFAs) are the most common malignant brain tumors in infants and have a poor prognosis.
- Epigenetic alterations are hallmarks of PFAs, suggesting potential therapeutic targets like histone deacetylase inhibitors (HDACi).
Observation:
- A 3-year-old male with recurrent PFA, confirmed as PFA subtype A by methylation profiling and exhibiting a BCL-6 corepressor mutation, was treated with multimodal therapy.
- The treatment regimen included craniospinal irradiation with a focal boost and concurrent administration of Vorinostat, a histone deacetylase inhibitor.
Findings:
- Serial imaging demonstrated a progressive decrease in tumor burden following the combined treatment.
- Nearly complete resolution of the ependymoma was observed at 15 months post-irradiation.
Implications:
- This case highlights the potential efficacy of histone deacetylase inhibitors, such as Vorinostat, in managing aggressive, recurrent posterior fossa ependymomas.
- Targeting epigenetic modifications represents a promising therapeutic strategy for carefully selected pediatric ependymoma patients.
