title: Comparison of two different conditioning regimens before autologous transplantation for children with high-risk neuroblastoma.
abstract: BACKGROUND
  Although high-dose chemotherapy (HDC) represents the standard of treatment for high-risk neuroblastoma (NBL), the most effective conditioning regimen still remains to be identified.
  
  
  METHODS
  Forty-one high-risk NBL entered into local protocol based on induction chemotherapy, surgery and HDC with either etoposide/thiotepa/cyclophophamide (ETC) or i.v. busulfan and L-PAM (Bu/L-PAM).
  
  
  RESULTS
  Thirty-seven patients underwent HDC; 29 with ETC and 8 with Bu/L-PAM. No toxic deaths were recorded. The 5-year progression-free survival (PFS) of patients given ETC was 21% (95% confidence interval CI (9-36%), while PFS for patients given Bu/L-PAM was 88% (95% CI=39-98%) (p<0.05). In multivariate analysis, treatment with the ETC regimen predicted progression/recurrence with a hazard ratio (HR) of 16.8 (p<0.05), as well as MYCN amplification which had an HR of 4.4 (p<0.05).
  
  
  CONCLUSIONS
  Although the number of studied cases is limited, our data suggest that in high-risk NBL the combination of Bu/L-PAM is superior to the ETC regimen.
authors.affiliation: Hematology/Oncology Department, Bambino Gesù Children Hospital, Rome, Italy. deioris@opbg.net
authors.forename: Maria Antonietta
authors.initials: MA
authors.lastname: DE Ioris
authors.forename: Benedetta
authors.initials: B
authors.lastname: Contoli
authors.forename: Alessandro
authors.initials: A
authors.lastname: Jenkner
authors.forename: Maria Debora
authors.initials: MD
authors.lastname: DE Pasquale
authors.forename: Annalisa
authors.initials: A
authors.lastname: Serra
authors.forename: Luigi
authors.initials: L
authors.lastname: DE Sio
authors.forename: Rosanna
authors.initials: R
authors.lastname: Pessolano
authors.forename: Maria Carmen
authors.initials: MC
authors.lastname: Garganese
authors.forename: Alessandro
authors.initials: A
authors.lastname: Crocoli
authors.forename: Teresa
authors.initials: T
authors.lastname: Corneli
authors.forename: Renata
authors.initials: R
authors.lastname: Boldrini
authors.forename: Aurora
authors.initials: A
authors.lastname: Castellano
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002066:Busulfan / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D018572:Disease-Free Survival; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D005784:Gene Amplification; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D000071447:N-Myc Proto-Oncogene Protein; D009447:Neuroblastoma / Q000188:drug therapy* / Q000235:genetics / Q000532:radiotherapy / Q000601:surgery*; D009687:Nuclear Proteins / Q000235:genetics; D015513:Oncogene Proteins / Q000235:genetics; D012307:Risk Factors; D013852:Thiotepa / Q000008:administration & dosage; D019172:Transplantation Conditioning / Q000379:methods*
chemical_list: C459801:MYCN protein, human; D000071447:N-Myc Proto-Oncogene Protein; D009687:Nuclear Proteins; D015513:Oncogene Proteins; D005047:Etoposide; D003520:Cyclophosphamide; D013852:Thiotepa; D002066:Busulfan; D008558:Melphalan