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

Björn M. van Geel; Bwee Tien Poll-The; Aad Verrips; Jaap Jan Boelens; Stephan Kemp; Marc Engelen

doi:10.1007/s10545-014-9797-1

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

Björn M. van Geel
, Bwee Tien Poll-The
, Aad Verrips
, Jaap Jan Boelens
, Stephan Kemp
, Marc Engelen^*

^*Corresponding author for this work

Research output: Contribution to journal › Article › Academic › peer-review

1 Citation (Scopus)

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.

Original language	English
Pages (from-to)	359-361
Number of pages	3
Journal	Journal of Inherited Metabolic Disease
Volume	38
Issue number	2
DOIs	https://doi.org/10.1007/s10545-014-9797-1
Publication status	Published - Mar 2015

Keywords

CHAIN FATTY-ACIDS
ADRENOMYELONEUROPATHY
COHORT

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97_1Final published version, 95.6 KBLicence: Taverne

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@article{8303dc23877b40c89ba33e6c009611a7,

title = "Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective study",

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.",

keywords = "CHAIN FATTY-ACIDS, ADRENOMYELONEUROPATHY, COHORT",

author = "\{van Geel\}, \{Bj{\"o}rn M.\} and Poll-The, \{Bwee Tien\} and Aad Verrips and Boelens, \{Jaap Jan\} and Stephan Kemp and Marc Engelen",

year = "2015",

month = mar,

doi = "10.1007/s10545-014-9797-1",

language = "English",

volume = "38",

pages = "359--361",

journal = "Journal of Inherited Metabolic Disease",

issn = "0141-8955",

publisher = "Springer Netherlands",

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TY - JOUR

T1 - Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy

T2 - a retrospective study

AU - van Geel, Björn M.

AU - Poll-The, Bwee Tien

AU - Verrips, Aad

AU - Boelens, Jaap Jan

AU - Kemp, Stephan

AU - Engelen, Marc

PY - 2015/3

Y1 - 2015/3

N2 - 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.

AB - 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.

KW - CHAIN FATTY-ACIDS

KW - ADRENOMYELONEUROPATHY

KW - COHORT

UR - https://www.scopus.com/pages/publications/84925482951

U2 - 10.1007/s10545-014-9797-1

DO - 10.1007/s10545-014-9797-1

M3 - Article

C2 - 25488625

AN - SCOPUS:84925482951

SN - 0141-8955

VL - 38

SP - 359

EP - 361

JO - Journal of Inherited Metabolic Disease

JF - Journal of Inherited Metabolic Disease

IS - 2

ER -

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

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Keywords

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