Haematopoietic stem cell transplantation in 12 patients with cerebral X-linked adrenoleukodystrophy
Abstract
Unlabelled In an attempt to elucidate prognostic factors, the data on 12 boys who underwent haematopoietic stem cell transplantation (HSCT) for cerebral X-linked adrenoleukodystrophy were evaluated. Two further patients received HSCT but died from transplantation-related complications. The data included neurological examination, neuropsychological testing and magnetic resonance imaging (MRI). Follow-up after HSCT was up to 5.5 years. Six patients showed a moderate to severe clinical deterioration after HSCT including two who died within 6 months. In this group, a MRI severity score of 10 or higher before HSCT was associated with severe impairment and a score of more than 12 was followed by rapid deterioration and death after HSCT. The presence of neurological symptoms before HSCT also affected prognosis. Six patients showed no deterioration in neurological or neuropsychological assessment after HSCT. Conclusion our data confirm that haematopoietic stem cell transplantation can stop the progress of demyelination when performed at a critical early stage of the disease. The prognosis in an individual patient for the clinical course after stem cell transplantation can in general be given based on the status before transplantation, although individual patients may show an unexpected course.
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The risk of developing cerebral disease is highest in childhood but remains present throughout life.
“The risk of developing cerebral disease is highest in childhood but remains present throughout life.1,29,33-39”
From International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy (2022) · cites this paper as [34]
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Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy
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Kühl, J.-S., Suarez, F., Gillett, G. T., Hemmati, P. G., Snowden, J. A., Stadler, M., Vuong, G. L., Aubourg, P., Köhler, W., & Arnold, R. (2017) · Brain · doi:10.1093/brain/awx016 · Cited by 78 · see citing works
Self-citation
- Authors
- Baumann, M., Korenke, C., Weddige-Diedrichs, A., Wilichowski, E., Hunneman, D., Wilken, B., Brockmann, K., Klingebiel, T., Niethammer, D., Kühl, J., Ebell, W., & Hanefeld, F.
- Affiliations
- University of Göttingen (DE)
- University Hospital Frankfurt (DE)
- University Children's Hospital Tübingen (DE)
- Humboldt-Universität zu Berlin (DE)
- Corresponding author
- Hanefeld, Folker
- Venue
- European Journal of Pediatrics
- Published
- 2003-01-01
- Volume
- 162
- Issue
- 1
- Pages
- 6-14
- ISSN
- 0340-6199 (ISSN-L)
- 1432-1076
- OpenAlex
- W1976333363
- Cited by
- 47
- References
- 6
- Citations by year
- 2025: 3 · 2024: 2 · 2023: 1 · 2022: 2 · 2021: 1 · 2019: 2 · 2018: 3 · 2017: 7 · 2016: 1 · 2014: 1 · 2013: 4 · 2012: 2
- FWCI
- 1.92
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- www.springer.com/tdm (tdm)
- Locations
- European Journal of Pediatrics (publishedVersion)
- PubMed (publishedVersion)
- GRO (submittedVersion)
- Topics
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Health Sciences › Medicine › Pathology and Forensic Medicine › Multiple Sclerosis Research Studies
- Keywords
- hematopoietic stem cell transplantation, prognosis, demyelination, neurological deterioration, neuropsychological outcomes, early-stage disease
- MeSH terms
- Adrenoleukodystrophy, Female, Follow-Up Studies, Humans, Magnetic Resonance Imaging, Neurologic Examination, Neuropsychological Tests, Treatment Outcome, Hematopoietic Stem Cell Transplantation
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