The experience of allogeneic hematopoietic stem cell transplantation in a patient with X-linked adrenoleukodystrophy.
Transfus Apher Sci
; 59(1): 102583, 2020 Feb.
Article
in 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.
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Transplantation, Homologous
/
Hematopoietic Stem Cell Transplantation
/
Adrenoleukodystrophy
/
Transplantation Conditioning
Limits:
Adult
/
Humans
/
Male
Language:
En
Journal:
Transfus Apher Sci
Journal subject:
HEMATOLOGIA
Year:
2020
Type:
Article