title: Treatment and prognostic factors in pleuropulmonary blastoma: an EXPeRT report.
abstract: BACKGROUND
  Pleuropulmonary blastoma (PPB) is an aggressive embryonal malignancy presenting in early childhood, presumably arising from pleuropulmonary mesenchyme. The European Cooperative Study Group for Paediatric Rare Tumours (EXPeRT) analysed its data on this tumour.
  
  
  METHODS
  This analysis concerns patients aged 0-17years with histologically-confirmed PPB registered up to 2008 in national databases in Italy, France and the United Kingdom and Poland. Lesions were classified as type I, II or III according to Dehner's classification.
  
  
  RESULTS
  Sixty-five patients were considered (13 type I, 24 type II and 28 type III). Most tumours were large (91% >5cm) and invaded the parietal pleura (29), mediastinum (10), major vessels (four) or pericardium (three). Regional nodes were involved in two cases, and three had metastases. The median follow-up was 5years (0.6-22). For type I patients, 5-year progression free survival (PFS) was 83.3% and overall survival 91.7%; six patients received no further treatment after surgery, but two relapsed. All type II/III PPB had chemotherapy (CT) and their 5-year PFS was 42.9% (27.7-57.2). On univariate analysis, favourable prognostic factors were: complete tumour resection at diagnosis (p=0.008); and absence of invasiveness (p=0.02); for type II/III tumours, type of CT was also a significant factor (patients given doxorubicin fared better, with a 5-year PFS of 70% versus 31.3% [p=0.01]).
  
  
  CONCLUSIONS
  Type I PPB patients' outcome was satisfactory. Complete resection at diagnosis seems important but rarely feasible for type II/III tumours, who benefited from doxorubicin-containing CT regimens. These results will inform the EXPeRT group's PPB treatment guidelines.
authors.affiliation: Pediatric Hematology and Oncology Division, Department of Woman's and Child's Health, Padova University Hospital, Padova, Italy. Electronic address: gianni.bisogno@unipd.it.
authors.forename: Gianni
authors.initials: G
authors.lastname: Bisogno
authors.affiliation: Department of Pediatric Oncology, Royal Manchester Children's Hospital, Manchester, United Kingdom.
authors.forename: Bernadette
authors.initials: B
authors.lastname: Brennan
authors.affiliation: Adolescent and Pediatric Oncology Unit, Institut Curie, 75005 Paris, France.
authors.forename: Daniel
authors.initials: D
authors.lastname: Orbach
authors.affiliation: Department of Pediatrics, Gdansk Medical University, Gdansk, Poland.
authors.forename: Teresa
authors.initials: T
authors.lastname: Stachowicz-Stencel
authors.affiliation: Pediatric Surgery Unit, Department of Woman's and Child's Health, Padova University Hospital, Padova, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Cecchetto
authors.affiliation: Pediatric Oncology Unit, Department of Pediatrics, II University, Napoli, Italy.
authors.forename: Paolo
authors.initials: P
authors.lastname: Indolfi
authors.affiliation: Department of Pediatrics, Gdansk Medical University, Gdansk, Poland.
authors.forename: Ewa
authors.initials: E
authors.lastname: Bien
authors.affiliation: Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milano, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Ferrari
authors.affiliation: Institut d'Hématologie et d'Oncologie Pédiatrique, Lyon, France.
authors.forename: Florence
authors.initials: F
authors.lastname: Dommange-Romero
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000494:pharmacology / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005060:Europe / Q000453:epidemiology; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D011379:Prognosis; D018202:Pulmonary Blastoma / Q000453:epidemiology / Q000473:pathology* / Q000628:therapy*; D035583:Rare Diseases / Q000453:epidemiology / Q000473:pathology / Q000628:therapy; D016019:Survival Analysis
keywords: Chemotherapy; Children; EXPeRT; Pleuropulmonary blastoma; TREP project; Very rare tumours