title: Peripheral medulloepithelioma: a rare tumor with a potential target therapy.
abstract: BACKGROUND
  Medulloepithelioma (ME) is a rare embryonal tumor predominantly located in the eye or in the central nervous system without an established treatment.
  
  
  METHODS
  We report of a case of a localized peripheral ME treated with conventional and high dose chemotherapy, surgery and local radiotherapy. At relapse, the tumor tissue revealed a different molecular signature compared to the initial tumor mass. This molecular signature revealed a high expression of platelet derived growth factor receptor (PDGFR). Sorafenib plus irinotecan and temozolomide was started with a 5 month progression free survival.
  
  
  CONCLUSIONS
  Our experience suggests a possible role of sorafenib or different PDGFR inhibitors in ME. Targeting treatment could represent an adjuvant and/or alternative therapy for ME and other rare tumors.
authors.affiliation: Department of Pediatric Hematology/Oncology and Stem Cell Transplantation, Bambino Gesù Children's Hospital, Piazza Sant'Onofrio, 4 - 00165 Rome, Italy. mdebora.depasquale@opbg.net.
authors.forename: Maria Debora
authors.initials: MD
authors.lastname: De Pasquale
authors.forename: Maria Antonietta
authors.initials: MA
authors.lastname: De Ioris
authors.forename: Angela
authors.initials: A
authors.lastname: Gallo
authors.forename: Angela
authors.initials: A
authors.lastname: Mastronuzzi
authors.forename: Alessandro
authors.initials: A
authors.lastname: Crocoli
authors.forename: Raffaele
authors.initials: R
authors.lastname: Cozza
authors.forename: Renata
authors.initials: R
authors.lastname: Boldrini
mesh_terms: D001932:Brain Neoplasms / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000473:pathology*; D002675:Child, Preschool; D017809:Fatal Outcome; D005260:Female; D006801:Humans; D007150:Immunohistochemistry; D058990:Molecular Targeted Therapy; D018242:Neuroectodermal Tumors, Primitive / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000473:pathology*; D014057:Tomography, X-Ray Computed