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Bone marrow transplant in adrenoleukodystrophy.

H W Moser, P J Tutschka, F R Brown

|November 1, 1984

Summary

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Allogeneic bone marrow transplant (BMT) normalized very long chain fatty acids (VLCFA) in a patient with adrenoleukodystrophy (ALD). However, neurological decline persisted, leading to death from adenovirus infection post-transplant.

Area of Science:

Biochemistry
Hematology
Genetics

Background:

Adrenoleukodystrophy (ALD) is a rare genetic disorder characterized by impaired very long chain fatty acid (VLCFA) metabolism.
ALD leads to progressive demyelination and neurological deterioration, particularly in males.
Allogeneic bone marrow transplant (BMT) is an experimental treatment option for ALD.

Observation:

A 13-year-old boy with rapidly progressive ALD underwent BMT from an HLA-identical sibling donor.
Hematologic engraftment and recovery were achieved within 4 weeks post-transplant.
Neurological deterioration continued despite successful engraftment.

Findings:

Biochemical markers of ALD, including white blood cell VLCFA levels and enzyme activity, normalized 10 days after BMT.

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Plasma VLCFA levels progressively reduced to near-normal ranges within 3 months.

The patient succumbed to an adenovirus infection 141 days after BMT.

Implications:

BMT can effectively correct the biochemical defect in ALD by restoring normal VLCFA metabolism.
Early BMT may be crucial for halting or slowing neurological progression in ALD.
Further research is needed to optimize BMT strategies and manage post-transplant complications like infections.