Busulfan-melphalan in high-risk neuroblastoma: the 30-year experience of a single institution.

Abstract:

:High-dose chemotherapy (HDC) was investigated in high-risk neuroblastoma (HR-NBL) to reduce the risk of relapse. We report the results of the 30-year experience of a cohort of patients with HR-NBL treated with high-dose (HD) busulfan (Bu)-containing regimens. From 1980 to 2009, 215 patients aged >1 year with stage 4 NBL were treated with HD Bu-containing regimens at Gustave Roussy. These data were prospectively recorded in the Pediatric Transplantation Database. The median age at diagnosis was 40 months (12-218 months). All patients had a stage 4 neuroblastoma. NMYC amplification was displayed in 24% of the tumors. The hematopoietic support consisted of bone marrow or PBSCs in 46% and 49% of patients, respectively. The 5-year event-free survival and overall survival rates of the whole cohort were 35.1% and 40%, respectively. Age at diagnosis, bone marrow involvement and tumor response after induction chemotherapy were significant prognostic factors. Toxicity was manageable and decreased over time, owing to both PBSC administration and better supportive care. Based on this experience, HD Bu-melphalan (Mel) has been implemented in Europe and compared with Carboplatin-Etoposide-Mel in the European SIOP Neuroblastoma (SIOPEN)/HR-NBL randomized protocol. It has now become the standard HDC in the SIOPEN HR strategy.

journal_name

Bone Marrow Transplant

authors

Proust-Houdemont S,Pasqualini C,Blanchard P,Dufour C,Benhamou E,Goma G,Semeraro M,Raquin MA,Hartmann O,Valteau-Couanet D

doi

10.1038/bmt.2016.75

subject

Has Abstract

pub_date

2016-08-01 00:00:00

pages

1076-81

issue

8

eissn

0268-3369

issn

1476-5365

pii

bmt201675

journal_volume

51

pub_type

杂志文章
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    authors: Favrot M,Philip T,Combaret V,Lemerle J,Marie JP,Bernard A

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    journal_title:Bone marrow transplantation

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    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

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    pub_type: 杂志文章,评审

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    doi:

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    doi:

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    doi:

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    doi:10.1038/sj.bmt.1700739

    authors: Mattijssen V,Schattenberg A,Schaap N,Preijers F,De Witte T

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    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704209

    authors: Dalle JH,Wall D,Theoret Y,Duval M,Shaw L,Larocque D,Taylor C,Gardiner J,Vachon MF,Champagne MA

    更新日期:2003-10-01 00:00:00

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    pub_type: 杂志文章,多中心研究,随机对照试验

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    authors: Biron P,Durand M,Roché H,Delozier T,Battista C,Fargeot P,Spaeth D,Bachelot T,Poiget E,Monnot F,Tanguy ML,Curé H

    更新日期:2008-03-01 00:00:00

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    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702145

    authors: De Bruyn C,Delforge A,Lagneaux L,Bron D

    更新日期:2000-02-01 00:00:00