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

Research output: Contribution to journalArticlepeer-review

90 Scopus citations


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 languageEnglish (US)
Pages (from-to)1410-1417
Number of pages8
Issue number11
StatePublished - Nov 1984

ASJC Scopus subject areas

  • Clinical Neurology


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