Related Experiment Video
Updated: Apr 19, 2026
Author Spotlight: Exploring the Lifespan Dynamics of Healthy Human Hematopoiesis
Published on: December 8, 2023
Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective
Björn M van Geel1, Bwee Tien Poll-The, Aad Verrips
1Department of Neurology, Medical Center Alkmaar, Alkmaar, The Netherlands.
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.
Related Concept Videos
01:22Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
01:21Stem Cell Therapy for Tissue Regeneration
Types of Stem Cells used in Stem Cell Therapy
The two main cell...