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Neuroblasstoma

Consolidation with a busulfan-containing regimen followed by stem cell transplantation in infants with poor prognosis stage 4 neuroblastoma

Bone Marrow Transplantation volume 25, pages 937–942 (2000)Cite this article

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.

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Acknowledgements

We are grateful to L Saint Ange for editing the manuscript, and to Severine Faure for her secretarial assistance.

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Authors and Affiliations

  1. Pediatrics Department, Institut Gustave Roussy, Villejuif, France

    D Valteau-Couanet, G Vassal & F Stambouli

  2. Biostatistics Department, Institut Gustave Roussy, Villejuif, France

    E Benhamou & O Hartmann

  3. Hematology Unit, Institut Gustave Roussy, Villejuif, France

    V Lapierre

  4. Radiology Department, Institut Gustave Roussy, Villejuif, France

    D Couanet

  5. Nuclear Medicine Department Institut Gustave Roussy, Villejuif, France

    J Lumbroso

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Valteau-Couanet, D., Benhamou, E., Vassal, G. et al. Consolidation with a busulfan-containing regimen followed by stem cell transplantation in infants with poor prognosis stage 4 neuroblastoma. Bone Marrow Transplant 25, 937–942 (2000). https://doi.org/10.1038/sj.bmt.1702376

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