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From the John F. Kennedy Institute (Drs Moser, Brown, A.E. Moser, Singh, and Ms. Mark) and the Oncology Center at Johns Hopkins Hospital (Drs. Tutschka, Yeager, and Santos); the Departments of Medicine (Drs. Tutachka, Yeager, and Santos), Neurology and Pediatrics (Drs. H.W. Moser, Brown, A.E. Moser, Singh, and Ms. Mark), Wilmer Ophthalmological Institute (Drs. Maumenee and Green), Pathology (Drs. McDonnell and White), and Radiology (Dr. Kumar), Johns Hopkins University, Baltimore, MD; and the Department of Pathology, Medical University of South Carolina, Charleston, SC (Dr. Powers).
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.
Address correspondence and reprint requests to Dr. H.W. Moser, John F. Kennedy Institute, 707 North Broadway, Baltimore, MD 21205.
Supported in part by Grants HD 10981, CA 06793, CA 15396, and EY 01773 from the US Public Health Service and BNS-83-09099 from the National Science Foundation. Dr. Frank Brown is a Joseph P. Kennedy, Jr., Scholar in Mental Retardation.
Accepted for publication April 13, 1984.
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