The experience of allogeneic hematopoietic stem cell transplantation in a patient with X-linked adrenoleukodystrophy.
Transfus Apher Sci
; 59(1): 102583, 2020 Feb.
Article
em En
| MEDLINE
| ID: mdl-31350055
ABSTRACT
X-linked adrenoleukodystrophy (X-ALD), a progressive neurometabolic disorder that is caused by a defect in the gene ABCD1 (ATP-binding cassette, subfamily D, member 1), which encodes the peroxisomal ABC half-transporter ALD protein. Recently, allogeneic hematopoietic stem cell transplantation (alloHSCT) is the only therapy known to prevent disease progression. In this study, we would like to present our experience of alloHSCT for X-ALD from a HLA matched related sibling by the use of reduced intensity conditioning regimen composed of fludarabine, busulfan and ATG which allows us to reduce procedure-related toxicity and prevent mortality while achieving a curative effect.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Contexto em Saúde:
6_ODS3_enfermedades_notrasmisibles
Base de dados:
MEDLINE
Assunto principal:
Transplante Homólogo
/
Transplante de Células-Tronco Hematopoéticas
/
Adrenoleucodistrofia
/
Condicionamento Pré-Transplante
Limite:
Adult
/
Humans
/
Male
Idioma:
En
Revista:
Transfus Apher Sci
Ano de publicação:
2020
Tipo de documento:
Article