title: Adverse outcome of infants with metastatic neuroblastoma, MYCN amplification and/or bone lesions: results of the French society of pediatric oncology.
abstract: To assess the relevance of MYCN amplification and bone lesions in stage 4 neuroblastoma (NB) in infants aged <1 year, 51 infants with stage 4 NB were enrolled. Three groups of patients were defined according to the type of metastases and the resectability of the primary tumour. Group I comprised 21 infants with radiologically detectable bone lesions, Group II 22 patients with an unresectable primary tumour and Group III eight patients with only metaiodobenzylguanidine (MIBG) skeletal uptake. MYCN oncogene content was assayed in 47/51 tumours and found to be amplified in 17 (37%). The 5-year event-free survival (EFS) rate of these 51 infants was 64.1% (+/- 7.1%). In a univariate analysis, bone lesions, MYCN amplification, urinary vanillylmandelic/homovanillic acid ratio and serum ferritin levels adversely influenced outcome. In the multivariate analysis, radiologically detectable bone lesions were the most powerful unfavourable prognostic indicator: the EFS rate was 27.2% for these infants compared to 90% for infants without bone lesions (P<0.0001). Our data emphasize the poor prognosis of infants affected by stage 4 NB with bone lesions, especially when associated with MYCN amplification. Given the poor results in this group whatever the treatment, new therapeutic approaches need to be investigated in the future.
authors.affiliation: Department of Pediatrics, Institut Gustave Roussy, 39 rue Camille Desmoulins, Villejuif Cedex, 94805, France.
authors.forename: V
authors.initials: V
authors.lastname: Minard
authors.forename: O
authors.initials: O
authors.lastname: Hartmann
authors.forename: M C
authors.initials: MC
authors.lastname: Peyroulet
authors.forename: J
authors.initials: J
authors.lastname: Michon
authors.forename: C
authors.initials: C
authors.lastname: Coze
authors.forename: A S
authors.initials: AS
authors.lastname: Defachelle
authors.forename: O
authors.initials: O
authors.lastname: Lejars
authors.forename: Y
authors.initials: Y
authors.lastname: Perel
authors.forename: C
authors.initials: C
authors.lastname: Bergeron
authors.forename: P
authors.initials: P
authors.lastname: Boutard
authors.forename: G
authors.initials: G
authors.lastname: Leverger
authors.forename: J L
authors.initials: JL
authors.lastname: Stephan
authors.forename: A
authors.initials: A
authors.lastname: Thyss
authors.forename: P
authors.initials: P
authors.lastname: Chastagner
authors.forename: G
authors.initials: G
authors.lastname: Couillault
authors.forename: C
authors.initials: C
authors.lastname: Devalck
authors.forename: P
authors.initials: P
authors.lastname: Lutz
authors.forename: F
authors.initials: F
authors.lastname: Mechinaud
authors.forename: F
authors.initials: F
authors.lastname: Millot
authors.forename: D
authors.initials: D
authors.lastname: Plantaz
authors.forename: X
authors.initials: X
authors.lastname: Rialland
authors.forename: H
authors.initials: H
authors.lastname: Rubie
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D001859:Bone Neoplasms / Q000401:mortality / Q000473:pathology / Q000556:secondary* / Q000628:therapy; D018572:Disease-Free Survival; D005260:Female; D005784:Gene Amplification; D016259:Genes, myc; D006719:Homovanillic Acid / Q000652:urine; D006801:Humans; D007223:Infant; D008297:Male; D009362:Neoplasm Metastasis; D009367:Neoplasm Staging; D009447:Neuroblastoma / Q000235:genetics / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D011379:Prognosis; D015996:Survival Rate; D016896:Treatment Outcome; D014642:Vanilmandelic Acid / Q000652:urine
chemical_list: D014642:Vanilmandelic Acid; D006719:Homovanillic Acid