Peripheral medulloepithelioma: A rare tumor with a potential target therapy

Research output: Contribution to journalArticlepeer-review


Background: Medulloepithelioma (ME) is a rare embryonal tumor predominantly located in the eye or in the central nervous system without an established treatment.Case presentation: 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.Conclusion: 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.

Original languageEnglish
Article number49
JournalJournal of Translational Medicine
Issue number1
Publication statusPublished - Feb 21 2014


  • PDGF
  • Peripheral medulloepithelioma
  • Target therapy
  • Treatment

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)
  • Medicine(all)


Dive into the research topics of 'Peripheral medulloepithelioma: A rare tumor with a potential target therapy'. Together they form a unique fingerprint.

Cite this