Conditioning regimen for allogeneic bone marrow transplantation in children with acquired bone marrow failure: fludarabine/melphalan vs. fludarabine/cyclophosphamide.
Journal
Bone marrow transplantation
ISSN: 1476-5365
Titre abrégé: Bone Marrow Transplant
Pays: England
ID NLM: 8702459
Informations de publication
Date de publication:
07 2020
07 2020
Historique:
received:
13
09
2019
accepted:
05
05
2020
revised:
01
05
2020
pubmed:
24
5
2020
medline:
22
6
2021
entrez:
24
5
2020
Statut:
ppublish
Résumé
Fludarabine/cyclophosphamide-based conditioning regimens are standard in bone marrow transplantation (BMT) for acquired bone marrow failure in children, however, graft failure may occur. Using the data from a nationwide transplantation registry, we compared the outcomes of children aged <16 years with acquired aplastic anemia and refractory cytopenia of childhood who underwent allogeneic BMT with either fludarabine/melphalan (n = 71) or fludarabine/cyclophosphamide (n = 296) between 2000 and 2016. The fludarabine/melphalan regimen provided excellent outcomes, with 3-year overall survival and failure-free survival rates of 98% and 97%, respectively. The 83% 3-year failure-free survival in the fludarabine/cyclophosphamide group was significantly inferior (P = 0.002), whereas the overall survival did not differ between the two groups. Late graft failure was the most common cause of treatment failure in the fludarabine/cyclophosphamide group, which experienced a significantly higher incidence of late graft failure than the fludarabine/melphalan group (11% vs. 3%; P = 0.035). Multivariate analyses showed that the fludarabine/melphalan regimen was associated with a better failure-free survival (hazard ratio [HR] 0.12; P = 0.005) and lower risk of late graft failure (HR 0.16; P = 0.037). Fludarabine/melphalan-based conditioning regimen can be a promising option for children with acquired bone marrow failure receiving BMT.
Identifiants
pubmed: 32444864
doi: 10.1038/s41409-020-0948-8
pii: 10.1038/s41409-020-0948-8
doi:
Substances chimiques
Cyclophosphamide
8N3DW7272P
Vidarabine
FA2DM6879K
fludarabine
P2K93U8740
Melphalan
Q41OR9510P
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
1272-1281Subventions
Organisme : Japan Agency for Medical Research and Development (AMED)
ID : 19ek0510023h0002
Pays : International
Investigateurs
Nao Yoshida
(N)
Yoshiyuki Takahashi
(Y)
Hiromasa Yabe
(H)
Ryoji Kobayashi
(R)
Ken-Ichiro Watanabe
(KI)
Kazuko Kudo
(K)
Keisuke Kato
(K)
Hideki Muramatsu
(H)
Atsushi Narita
(A)
Manabu Wakamatsu
(M)
Seiji Kojima
(S)
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