Recommendations on hematopoietic stem cell transplantation for patients with Diamond-Blackfan anemia. On behalf of the Pediatric Diseases and Severe Aplastic Anemia Working Parties of the EBMT.
Journal
Bone marrow transplantation
ISSN: 1476-5365
Titre abrégé: Bone Marrow Transplant
Pays: England
ID NLM: 8702459
Informations de publication
Date de publication:
12 2021
12 2021
Historique:
received:
01
06
2021
accepted:
20
08
2021
revised:
09
08
2021
pubmed:
1
9
2021
medline:
11
3
2022
entrez:
31
8
2021
Statut:
ppublish
Résumé
Diamond Blackfan anemia (DBA) is a rare congenital syndrome presenting primarily as pure red cell aplasia with constitutional abnormalities and cancer predisposition. Established treatment options are corticosteroids, regular erythrocyte transfusions with iron chelation therapy, and hematopoietic stem cell transplantation (HSCT). To date, HSCT is the only definitive curative treatment for the hematological phenotype of DBA, but there is little experience with its use. Given the rarity of the disease and its unique features, an expert panel agreed to draw up a set of recommendations on the use of HSCT in DBA to guide clinical decision-making and practice. The recommendations address indications, pretransplant patient evaluation, donor selection, stem cell sources, conditioning regimens, prophylaxis of rejection and graft versus host disease, and post-transplant follow-up.
Identifiants
pubmed: 34462566
doi: 10.1038/s41409-021-01449-w
pii: 10.1038/s41409-021-01449-w
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
2956-2963Informations de copyright
© 2021. The Author(s), under exclusive licence to Springer Nature Limited.
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