Bone marrow transplant in adrenoleukodystrophy

Hugo W. Moser; Peter J. Tutschka; Frank R. Brown; Ann E. Moser; Andrew M. Yeager; Inderjit Singh; Shirley A. Mark; Ashok A.J. Kumar; Jan M. McDonnell; Charles L. White; Irene H. Maumenee; William R. Green; James M. Powers; George W. Santos

doi:10.1212/wnl.34.11.1410

Bone marrow transplant in adrenoleukodystrophy

Hugo W. Moser, Peter J. Tutschka, Frank R. Brown, Ann E. Moser, Andrew M. Yeager, Inderjit Singh, Shirley A. Mark, Ashok A.J. Kumar, Jan M. McDonnell, Charles L. White, Irene H. Maumenee, William R. Green, James M. Powers, George W. Santos

CCSG - Brain Cancer

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

90 Scopus citations

Abstract

An allogeneic bone marrow transplant (BMT) from a normal HLA identical sibling donor was performed in a 13-year-old boy with rapidly progressive adrenoleukodystrophy (ALD). Engraftment and complete hematologic recovery occurred within 4 weeks, but neurologic deterioration continued. The patient died of an adenovirus infection 141 days after BMT. ALD is characterized by abnormally high plasma levels of very long chain fatty acids (VLCFA) as a result of impaired capacity to degrade them. Ten days after BMT, the white blood cell VLCFA levels and enzyme activity became normal; after 3 months, there was progressive reduction of plasma VLCFA to levels only slightly above normal.

Original language	English (US)
Pages (from-to)	1410-1417
Number of pages	8
Journal	Neurology
Volume	34
Issue number	11
DOIs	https://doi.org/10.1212/wnl.34.11.1410
State	Published - Nov 1984

ASJC Scopus subject areas

Clinical Neurology

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@article{3164132da7764b509b621ab2dd674d9e,

title = "Bone marrow transplant in adrenoleukodystrophy",

abstract = "An allogeneic bone marrow transplant (BMT) from a normal HLA identical sibling donor was performed in a 13-year-old boy with rapidly progressive adrenoleukodystrophy (ALD). Engraftment and complete hematologic recovery occurred within 4 weeks, but neurologic deterioration continued. The patient died of an adenovirus infection 141 days after BMT. ALD is characterized by abnormally high plasma levels of very long chain fatty acids (VLCFA) as a result of impaired capacity to degrade them. Ten days after BMT, the white blood cell VLCFA levels and enzyme activity became normal; after 3 months, there was progressive reduction of plasma VLCFA to levels only slightly above normal.",

author = "Moser, {Hugo W.} and Tutschka, {Peter J.} and Brown, {Frank R.} and Moser, {Ann E.} and Yeager, {Andrew M.} and Inderjit Singh and Mark, {Shirley A.} and Kumar, {Ashok A.J.} and McDonnell, {Jan M.} and White, {Charles L.} and Maumenee, {Irene H.} and Green, {William R.} and Powers, {James M.} and Santos, {George W.}",

year = "1984",

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T1 - Bone marrow transplant in adrenoleukodystrophy

AU - Moser, Hugo W.

AU - Tutschka, Peter J.

AU - Brown, Frank R.

AU - Moser, Ann E.

AU - Yeager, Andrew M.

AU - Singh, Inderjit

AU - Mark, Shirley A.

AU - Kumar, Ashok A.J.

AU - McDonnell, Jan M.

AU - White, Charles L.

AU - Maumenee, Irene H.

AU - Green, William R.

AU - Powers, James M.

AU - Santos, George W.

PY - 1984/11

Y1 - 1984/11

N2 - An allogeneic bone marrow transplant (BMT) from a normal HLA identical sibling donor was performed in a 13-year-old boy with rapidly progressive adrenoleukodystrophy (ALD). Engraftment and complete hematologic recovery occurred within 4 weeks, but neurologic deterioration continued. The patient died of an adenovirus infection 141 days after BMT. ALD is characterized by abnormally high plasma levels of very long chain fatty acids (VLCFA) as a result of impaired capacity to degrade them. Ten days after BMT, the white blood cell VLCFA levels and enzyme activity became normal; after 3 months, there was progressive reduction of plasma VLCFA to levels only slightly above normal.

AB - An allogeneic bone marrow transplant (BMT) from a normal HLA identical sibling donor was performed in a 13-year-old boy with rapidly progressive adrenoleukodystrophy (ALD). Engraftment and complete hematologic recovery occurred within 4 weeks, but neurologic deterioration continued. The patient died of an adenovirus infection 141 days after BMT. ALD is characterized by abnormally high plasma levels of very long chain fatty acids (VLCFA) as a result of impaired capacity to degrade them. Ten days after BMT, the white blood cell VLCFA levels and enzyme activity became normal; after 3 months, there was progressive reduction of plasma VLCFA to levels only slightly above normal.

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JO - Neurology

JF - Neurology

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Bone marrow transplant in adrenoleukodystrophy

Abstract

ASJC Scopus subject areas

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