Ongoing Response in a Multiply Relapsed Metastatic Posterior Fossa Ependymoma A After Vorinostat and Concomitant Irradiation. Issue 2 (March 2022)

• Record Type: Journal Article
• Authors: Gorsi, Hamza S.; Toll, Stephanie A.; Sood, Sandeep; Miler, Steven; Altinok, Deniz; Kumar-Sinha, Chandan; Mody, Rajen; Yankelevich, Maxim
• Is Part Of: Journal of pediatric hematology/oncology. Volume 44:Issue 2(2022)
• Publication Date: 2022-03
• Subjects: posterior fossa ependymoma A -- HDAC inhibitor -- vorinostat -- epigenetic modifier; Pediatric hematology -- Periodicals; Tumors in children -- Periodicals; 618.9215
• DOI: 10.1097/MPH.0000000000002175
• Languages: English
• ISSNs: 1077-4114

Abstract

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.