Hematopoietic Stem Cell Transplantation is a cost-effective alternative to enzyme replacement therapy in Gaucher Disease.
Gaucher disease (GD)
Hematopoietic stem cell Transplantation (HSCT)
enzyme replacement therapy (ERT)
Journal
Blood cell therapy
ISSN: 2432-7026
Titre abrégé: Blood Cell Ther
Pays: Japan
ID NLM: 9918333884906676
Informations de publication
Date de publication:
25 Aug 2022
25 Aug 2022
Historique:
received:
14
09
2021
accepted:
09
01
2022
entrez:
30
1
2023
pubmed:
31
1
2023
medline:
31
1
2023
Statut:
epublish
Résumé
Allogeneic hematopoietic stem cell transplantation (HSCT) is a feasible treatment option for Gaucher disease (GD). Among 60 patients diagnosed with GD over 15 years (2004-2019), three children who underwent HSCT (January-November 2017) were analyzed. Two boys (cases 1 and 2) and one girl (case 3) received HSCT at 3, 7, and 10 years of age, respectively. Cases 1 and 3 received haplo-HSCT, while case 2 received HLA-identical related-donor transplantation. The CD 34 cell dose was 5-10×10
Identifiants
pubmed: 36712555
doi: 10.31547/bct-2021-020
pmc: PMC9873422
doi:
Types de publication
Journal Article
Langues
eng
Pagination
69-74Informations de copyright
Copyright Ⓒ2022 Asia-Pacific Blood and Marrow Transplantation Group (APBMT).
Déclaration de conflit d'intérêts
The authors declare no conflicts of interest. Disclosure forms provided by the authors are available on the website. AS is a member of the Editor of Blood Cell Therapy. He is not involved in the editorial evaluation or the decision to accept this article for publication.
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