title: Characteristics and outcome of patients with ganglioneuroblastoma, nodular subtype: a report from the INRG project.
abstract: OBJECTIVE
  Describe characteristics and outcome of INRG patients with ganglioneuroblastoma, nodular subtype (GNBn).
  
  
  METHODS
  Amongst 4071 patients in the INRG database with known INPC histological category, 232 patients with GNBn were identified. Patients were categorised by clinical, pathological and genetic characteristic. For event-free survival (EFS) and overall survival (OS), Kaplan-Meier curves and lifetables were generated, and the outcome of subgroups was compared using log rank test.
  
  
  RESULTS
  Patients with GNBn were older (83% >18 months), a higher proportion had unfavourable INPC pathology (83%), and rarely had MYCN gene amplified tumours (2%). Otherwise, the distribution of clinical and biological risk factors including stage, ferritin, initial treatment, grade of NB differentiation, MKI, 11q, 1p, and 17q were similar between patients with GNBn and the overall INRG cohort. EFS and OS were 54%±5% and 68%±5%, respectively. A cohort with superior outcome was identified: OS for GNBn patients younger than 18 months was 95%±5% (n=39) and for GNBn patients with stage 1, 2, 3, 4s was 95%±3% (n=125). Conversely, a poor outcome sub-group could also be identified: OS for stage 4 was 35%±7% (n=107).
  
  
  CONCLUSIONS
  Patients with GNBn tumours are rare and have a very heterogeneous outcome. Except for LDH and MKI, the factors prognostic in the overall NB cohort are also prognostic in patients with GNBn. Similar to the overall NB cohort, patients with GNBn older than 18 months of age, with stage 4 disease represent a high-risk sub-group and should be considered for aggressive treatment upfront.
authors.affiliation: Department of Paediatrics, Division of Haematology and Oncology, The Hospital for Sick Children, 555 University Avenue, Toronto, Ontario, Canada.
authors.forename: Paola
authors.initials: P
authors.lastname: Angelini
authors.forename: Wendy B
authors.initials: WB
authors.lastname: London
authors.forename: Susan L
authors.initials: SL
authors.lastname: Cohn
authors.forename: Andrew D J
authors.initials: AD
authors.lastname: Pearson
authors.forename: Katherine K
authors.initials: KK
authors.lastname: Matthay
authors.forename: Tom
authors.initials: T
authors.lastname: Monclair
authors.forename: Peter F
authors.initials: PF
authors.lastname: Ambros
authors.forename: Hiroyuki
authors.initials: H
authors.lastname: Shimada
authors.forename: Ivo
authors.initials: I
authors.lastname: Leuschner
authors.forename: Michel
authors.initials: M
authors.lastname: Peuchmaur
authors.forename: Meredith S
authors.initials: MS
authors.lastname: Irwin
authors.forename: Sylvain
authors.initials: S
authors.lastname: Baruchel
mesh_terms: D000367:Age Factors; D018305:Ganglioneuroblastoma / Q000235:genetics / Q000401:mortality* / Q000473:pathology; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D000071447:N-Myc Proto-Oncogene Protein; D009367:Neoplasm Staging; D009687:Nuclear Proteins / Q000235:genetics; D015513:Oncogene Proteins / Q000235:genetics; D011003:Ploidies
chemical_list: C459801:MYCN protein, human; D000071447:N-Myc Proto-Oncogene Protein; D009687:Nuclear Proteins; D015513:Oncogene Proteins