title: Consolidation with a busulfan-containing regimen followed by stem cell transplantation in infants with poor prognosis stage 4 neuroblastoma.
abstract: Although infants with stage 4 neuroblastoma (NB) usually have a good prognosis, metastatic relapses after 1 year of age and amplification of the N-myc oncogene are established poor prognostic factors. In order to improve the survival of patients with such high-risk factors, we performed consolidation with a busulfan (600 mg/m2)-melphalan (140 mg/m2)-containing regimen followed by autologous stem cell transplantation (SCT). From 1986 to 1998, 12 patients were treated according to this strategy. Their median age at diagnosis was 9 months (1-11). Consolidation was performed after a metastatic relapse in five children, because of persistent bone metastases in one and as first-line consolidation in six patients whose tumor exhibited N-myc amplification. The 5-year EFS rate is 64. 5% (36-85%) with a median follow-up of 92 months (20-126). One toxicity-related death occurred in a very heavily pretreated patient. Hepatic veno-occlusive disease was the major side-effect that occurred in nine of 12 children. This busulfan-melphalan combination appears to dramatically improve the prognosis of these high-risk infants with metastatic NB. Given its high toxicity, indications for this consolidation must be restricted to high-risk infants and a lower dose of busulfan (480 mg/m2) is recommended in children weighing less than 10 kg. Bone Marrow Transplantation (2000) 25, 937-942.
authors.affiliation: Pediatrics Department, Institut Gustave Roussy, Villejuif, France.
authors.forename: D
authors.initials: D
authors.lastname: Valteau-Couanet
authors.forename: E
authors.initials: E
authors.lastname: Benhamou
authors.forename: G
authors.initials: G
authors.lastname: Vassal
authors.forename: F
authors.initials: F
authors.lastname: Stambouli
authors.forename: V
authors.initials: V
authors.lastname: Lapierre
authors.forename: D
authors.initials: D
authors.lastname: Couanet
authors.forename: J
authors.initials: J
authors.lastname: Lumbroso
authors.forename: O
authors.initials: O
authors.lastname: Hartmann
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage*; D001835:Body Weight; D001932:Brain Neoplasms / Q000473:pathology / Q000628:therapy*; D002066:Busulfan / Q000008:administration & dosage*; D003131:Combined Modality Therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D007223:Infant; D008558:Melphalan / Q000008:administration & dosage; D009447:Neuroblastoma / Q000473:pathology / Q000628:therapy*; D011379:Prognosis; D016019:Survival Analysis; D014182:Transplantation, Autologous
chemical_list: D007166:Immunosuppressive Agents; D002066:Busulfan; D008558:Melphalan