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J Inherit Metab Dis. 2015 Mar;38(2):359-61. doi: 10.1007/s10545-014-9797-1. Epub 2014 Dec 9.

Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective study.

Author information

1
Department of Neurology, Medical Center Alkmaar, Alkmaar, The Netherlands.

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 AND METHODS:

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.

PMID:
25488625
DOI:
10.1007/s10545-014-9797-1
[Indexed for MEDLINE]

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