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- Title
Consolidation with a busulfan-containing regimen followed by stem cell transplantation in infants with poor prognosis stage 4 neuroblastoma.
- Authors
Valteau-Couanet, D; Benhamou, E; Vassal, G; Stambouli, F; Lapierre, V; Couanet, D; Lumbroso, J; Hartmann, O
- 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.
- Subjects
NEUROBLASTOMA; MYC oncogenes; STEM cell transplantation
- Publication
Bone Marrow Transplantation, 2000, Vol 25, Issue 9, p937
- ISSN
0268-3369
- Publication type
Article
- DOI
10.1038/sj.bmt.1702376