title: Embryonal tumors with multilayered rosettes in children: the SFCE experience.
abstract: OBJECTIVE
  The purpose of this study was to retrospectively study embryonal tumors with multilayered rosettes (ETMR), a rare new entity that gathers ETAN-TR (embryonal tumor with abundant neuropil and true rosettes), ependymoblastomas, and medulloepitheliomas, in order to improve their descriptions and try to better define therapeutic modalities.
  
  
  METHODS
  Patients with ETMR, ETAN-TR, ependymoblastoma, and medulloepithelioma treated in SFCE centres (Société Française de lutte contre les Cancers et les leucémies de l'Enfant et de l'adolescent) since 2000 were collected. Data were retrieved from clinical charts.
  
  
  RESULTS
  Thirty-eight patients were included in the analysis. Seventeen had an ETAN-TR, 13 had a medulloepithelioma, and 8 had an ETMR. No ependymoblastoma was included. The median age at diagnosis was 31 months (range, 2.8-141 months). The predominant tumor location was supratentorial (66%); 18.4% patients had metastatic lesion. LIN28A expression was positive in 11/11 patients. Amplification of the locus 19q13.42 was positive in 10/12 patients. Thirty patients were treated according to the primitive neuroectodermal tumors of high risk (PNET-HR) protocol. The median time of follow-up was 0.9 years (range 0.1 to 15.3 years). The 1-year event-free survival (EFS) and overall survival (OS) were, respectively, 36% CI 95% (23-55) and 45% CI 95% (31-64). On multivariate analysis, complete surgical resection, radiotherapy, and high-dose chemotherapy were associated with a better overall survival with a relative risk of, respectively, 7.9 CI 95% (2.6-23.5) p < 0.0002, 41.8 CI 95% (9.4-186) p < 0.0001, and 3.5 CI 95% (1.3-9.5) p = 0.012.
  
  
  CONCLUSIONS
  Prognosis of ETMR remains dismal despite multimodal therapy. LIN28A immunostaining and 19q13.42 amplification should be systematically done to secure the diagnosis. Complete surgical resection, radiotherapy, and high-dose chemotherapy are associated with better outcome.
authors.affiliation: Department of Pediatric Hematology-Oncology, APHM, La Timone Hospital, 264 rue Saint-Pierre, 13005, Marseille, France. meryl.horwitz@gmail.com.
authors.forename: Meryl
authors.initials: M
authors.lastname: Horwitz
authors.affiliation: Department of Pediatric and Adolescent Oncology, Gustave Roussy Campus, 114, rue Edouard-Vaillant, 94805, Villejuif Cedex, France. christelle.dufour@gustaveroussy.fr.
authors.forename: Christelle
authors.initials: C
authors.lastname: Dufour
authors.affiliation: Department of Pediatric Oncology, Oscar Lambret, 3 rue Frederic Combemale, BP 307, 59020, Lille Cedex, France. p-leblond@o-lambret.fr.
authors.forename: Pierre
authors.initials: P
authors.lastname: Leblond
authors.affiliation: Department of Pediatric Oncology, Institut Curie, 26, rue d'Ulm, 75005, Paris, France. franck.bourdeaut@curie.fr.|INSERM U830, Laboratory of Genetics and Cancer Biology, Institut Curie, Paris, France. franck.bourdeaut@curie.fr.
authors.forename: Franck
authors.initials: F
authors.lastname: Bourdeaut
authors.affiliation: Department of Pediatrics, Centre Léon Bérard, 28 Prom. Léa et Napoléon Bullukian, 69008, Lyon, France. cecile.conter@ihope.fr.
authors.forename: Cécile
authors.initials: C
authors.lastname: Faure-Conter
authors.affiliation: Department of Hematology-Oncology, Children University Hospital, 330 Avenue de Grande Bretagne, 31300, Toulouse, France. bertozzi.ai@chu-toulouse.fr.
authors.forename: Anne-Isabelle
authors.initials: AI
authors.lastname: Bertozzi
authors.affiliation: Department of Neuropathology, Toulouse Rangueil University Hospital, 1, avenue du Pr Jean Poulhès, 31400, Toulouse, France. delisle.b@chu-toulouse.fr.
authors.forename: Marie Bernadette
authors.initials: MB
authors.lastname: Delisle
authors.affiliation: Department of Pediatric Hematology-Oncology, University Hospital of Montpellier, 191, avenue du doyen Gaston Giraud, 34295, Montpellier cedex, France. gillespalen@yahoo.fr.
authors.forename: Gilles
authors.initials: G
authors.lastname: Palenzuela
authors.affiliation: Department of Pathology and neuropathology, "Pierre Werthmeier" Hospital, Boulevard Pinel, 69677, Bron, France. anne.jouvet@chu-lyon.fr.
authors.forename: Anne
authors.initials: A
authors.lastname: Jouvet
authors.affiliation: Department of Neurosurgery, APHM, La Timone Hospital, 264 rue Saint-Pierre, 13005, Marseille, France. didier.scavarda@ap-hm.fr.
authors.forename: Didier
authors.initials: D
authors.lastname: Scavarda
authors.affiliation: Department of Pediatric Neurosurgery, Lille University Hospital, 2 Avenue Oscar Lambret, 59000, Lille, France. matthieu.vinchon@chru-lille.fr.
authors.forename: Matthieu
authors.initials: M
authors.lastname: Vinchon
authors.affiliation: Department of Radiation Oncology and Pediatrics, APHM, La Timone Hospital, 264 rue Saint-Pierre, 13005, Marseille, France. laetitia.padovani@ap-hm.fr.
authors.forename: Laetitia
authors.initials: L
authors.lastname: Padovani
authors.affiliation: BIOSTIC, Pôle de Santé Publique, AP-HM, Marseille, 264 rue Saint-Pierre, 13005, Marseille, France. jean.gaudart@ap-hm.fr.|UMR912 SESSTIM, Aix-Marseille University, Marseille, France. jean.gaudart@ap-hm.fr.
authors.forename: Jean
authors.initials: J
authors.lastname: Gaudart
authors.affiliation: Department of Pathology, APHM, La Timone Hospital, Université Aix-Marseille, 264 rue Saint-Pierre, 13005, Marseille, France. dominiqueFrance.figarella@ap-hm.fr.
authors.forename: Dominique Figarella
authors.initials: DF
authors.lastname: Branger
authors.affiliation: Department of Pediatric Hematology-Oncology, APHM, La Timone Hospital, 264 rue Saint-Pierre, 13005, Marseille, France. nicolas.andre@ap-hm.fr.|INSERM UMR 911, Centre de Recherche en Oncologie biologique et en Oncopharmacologie, Aix-Marseille University, 264 rue Saint-Pierre, 13005, Marseille, France. nicolas.andre@ap-hm.fr.|Department of Pediatric Hematology and Oncology, Children Hospital of La Timone, 264, rue Saint-Pierre, 13005, Marseille, France. nicolas.andre@ap-hm.fr.
authors.forename: Nicolas
authors.initials: N
authors.lastname: Andre
mesh_terms: D001932:Brain Neoplasms / Q000473:pathology / Q000628:therapy*; D017024:Chemotherapy, Adjuvant; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D015999:Multivariate Analysis; D009373:Neoplasms, Germ Cell and Embryonal / Q000473:pathology / Q000628:therapy; D018242:Neuroectodermal Tumors, Primitive / Q000473:pathology / Q000628:therapy*; D019581:Neuropil / Q000473:pathology; D019635:Neurosurgical Procedures; D011379:Prognosis; D018714:Radiotherapy, Adjuvant; D012189:Retrospective Studies; D013120:Spinal Cord Neoplasms / Q000473:pathology / Q000628:therapy*
keywords: Brain tumors; Chemotherapy; Children; ETMR; Radiotherapy; Surgery