title: Pancreatoblastoma: a report from the European cooperative study group for paediatric rare tumours (EXPeRT).
abstract: BACKGROUND
  Pancreatoblastoma is a very rare malignant tumour typically occurring in the early years of life. Due to its rarity, standardised diagnostic and therapeutic guidelines are not available for pancreatoblastoma.
  
  
  METHODS
  The newborn cooperative group denominated EXPeRT - European cooperative study group for paediatric rare tumours - combined in a joint analysis of all cases registered between 2000 and 2009 by the national groups of Italy, France, United Kingdom, Poland and Germany.
  
  
  RESULTS
  Twenty patients <18years old (median age 4years) were analysed: nine had distant metastases at diagnosis. Seventeen patients had tumour resection, at initial or delayed surgery. Eighteen received chemotherapy (response rate 73%), seven received radiotherapy. For the whole series, 5-year event-free survival and overall survival were 58.8% and 79.4%, respectively. Outcome did not correlate with tumour site and size, but was strongly influenced by the feasibility of tumour complete resection.
  
  
  CONCLUSIONS
  This international study confirms the rarity of the disease, the critical role of surgical resection both as therapy and as a prognostic variable, and the potential efficacy of chemotherapy. The adoption of an intensive multidisciplinary approach is required, as well as the referral to highly experienced centres. Further international cooperation is needed to collect larger series and stimulate biological studies to improve our understanding of the biology and the natural history of PBL.
authors.affiliation: Department of Pediatrics, Medical University, Gdansk, Poland.
authors.forename: Ewa
authors.initials: E
authors.lastname: Bien
authors.forename: Jan
authors.initials: J
authors.lastname: Godzinski
authors.forename: Patrizia
authors.initials: P
authors.lastname: Dall'igna
authors.forename: Anne-Sophie
authors.initials: AS
authors.lastname: Defachelles
authors.forename: Teresa
authors.initials: T
authors.lastname: Stachowicz-Stencel
authors.forename: Daniel
authors.initials: D
authors.lastname: Orbach
authors.forename: Gianni
authors.initials: G
authors.lastname: Bisogno
authors.forename: Giovanni
authors.initials: G
authors.lastname: Cecchetto
authors.forename: Steven
authors.initials: S
authors.lastname: Warmann
authors.forename: Verena
authors.initials: V
authors.lastname: Ellerkamp
authors.forename: Bernadette
authors.initials: B
authors.lastname: Brennan
authors.forename: Anna
authors.initials: A
authors.lastname: Balcerska
authors.forename: Malgorzata
authors.initials: M
authors.lastname: Rapala
authors.forename: Ines
authors.initials: I
authors.lastname: Brecht
authors.forename: Dominik
authors.initials: D
authors.lastname: Schneider
authors.forename: Andrea
authors.initials: A
authors.lastname: Ferrari
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D017024:Chemotherapy, Adjuvant; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005060:Europe; D005260:Female; D006801:Humans; D007223:Infant; D053208:Kaplan-Meier Estimate; D008297:Male; D010180:Pancreatectomy; D010190:Pancreatic Neoplasms / Q000401:mortality / Q000473:pathology / Q000628:therapy; D018714:Radiotherapy, Adjuvant; D035583:Rare Diseases; D015996:Survival Rate; D013997:Time Factors; D016896:Treatment Outcome