Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective study.
J Inherit Metab Dis
; 38(2): 359-61, 2015 Mar.
Article
em En
| MEDLINE
| ID: mdl-25488625
ABSTRACT
BACKGROUND:
X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal metabolic disorder. Male patients develop adrenocortical insufficiency (80 % before 18 years), a chronic myelopathy (adrenomyeloneuropathy (AMN); all in adulthood), or progressive cerebral demyelination (cerebral ALD; 40 % before 18 years). Cerebral ALD is treated with haematopoetic cell transplantation (HCT). It is unknown if AMN still develops in patients with X-ALD that underwent HCT for cerebral ALD in childhood. PATIENTS ANDMETHODS:
A retrospective observational study was performed by selecting all adult patients with X-ALD in our cohort that underwent HCT in childhood.RESULTS:
This retrospective study found that three out of five patients in our cohort who underwent HCT in childhood developed signs of myelopathy in adulthood.CONCLUSION:
These data suggest that HCT for cerebral ALD in childhood does not prevent the onset of AMN in X-ALD in adulthood.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Doenças da Medula Espinal
/
Transplante de Células-Tronco Hematopoéticas
/
Adrenoleucodistrofia
Idioma:
En
Ano de publicação:
2015
Tipo de documento:
Article
País de afiliação:
Holanda