Myeloablative megatherapy with autologous stem-cell rescue versus oral maintenance chemotherapy as consolidation treatment in patients with high-risk neuroblastoma: a randomised controlled trial.

Berthold, F; Boos, J; Burdach, S; Erttmann, R; Henze, G; Hermann, J; Klingebiel, T; Kremens, B; Schilling, FH; Schrappe, M; Simon, T; Hero, B

doi:10.1016/S1470-2045(05)70291-6

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Title:: Myeloablative megatherapy with autologous stem-cell rescue versus oral maintenance chemotherapy as consolidation treatment in patients with high-risk neuroblastoma: a randomised controlled trial.
Document type:: Clinical Trial; Journal Article; Multicenter Study; Randomized Controlled Trial; Article
Author(s):: Berthold, F; Boos, J; Burdach, S; Erttmann, R; Henze, G; Hermann, J; Klingebiel, T; Kremens, B; Schilling, FH; Schrappe, M; Simon, T; Hero, B
Abstract:: BACKGROUND: Myeloablative megatherapy is commonly used to improve the poor outlook of children with high-risk neuroblastoma, yet its role is poorly defined. We aimed to assess whether megatherapy with autologous stem-cell transplantation could increase event-free survival and overall survival compared with maintenance chemotherapy. METHODS: 295 patients with high-risk neuroblastoma (ie, patients with stage 4 disease aged older than 1 year or those with MYCN-amplified tumours and stage 1, 2, 3, or 4S disease or stage 4 disease and <1 year old) were randomly assigned to myeloablative megatherapy (melphalan, etoposide, and carboplatin) with autologous stem-cell transplantation (n=149) or to oral maintenance chemotherapy with cyclophosphamide (n=146). The primary endpoint was event-free survival. Secondary endpoints were overall survival and the number of treatment-related deaths. Analyses were done by intent to treat, as treated, and treated as randomised. FINDINGS: Intention-to-treat analysis showed that patients allocated megatherapy had increased 3-year event-free survival compared with those allocated maintenance therapy (47% [95% CI 38-55] vs 31% [95% CI 23-39]; hazard ratio 1.404 [95% CI 1.048-1.881], p=0.0221), but did not have significantly increased 3-year overall survival (62% [95% CI 54-70] vs 53% [95% CI 45-62]; 1.329 [0.958-1.843], p=0.0875). Improved 3-year event-free survival and 3-year overall survival were also recorded for patients given megatherapy in the as-treated group (n=212) and in the treated-as-randomised group (n=145). Two patients died from therapy-related complications during induction treatment. No patients given maintenance therapy died from acute treatment-related toxic effects. Five patients given megatherapy died from acute complications related to megatherapy. INTERPRETATION: Myeloablative chemotherapy with autologous stem-cell transplantation improves the outcome for children with high-risk neuroblastoma despite the raised risk of treatment-associated death.
Journal title abbreviation:: Lancet Oncol
Year:: 2005
Journal volume:: 6
Journal issue:: 9
Pages contribution:: 649-58
Language:: eng
Fulltext / DOI:: doi:10.1016/S1470-2045(05)70291-6
Pubmed ID:: http://view.ncbi.nlm.nih.gov/pubmed/16129365
Print-ISSN:: 1470-2045
TUM Institution:: Klinik und Poliklinik für Kinderheilkunde und Jugendmedizin
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mediaTUM Gesamtbestand Einrichtungen Schools TUM School of Medicine and Health Departments Clinical Medicine Klinik und Poliklinik für Kinder- und Jugendmedizin (Prof. Hauer)2005