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:
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Authors | Matthias Baumann, G Christoph Korenke, Almuth Weddige-Diedrichs, Ekkehard Wilichowski, Donald H Hunneman, Bernd Wilken, Knut Brockmann, Thomas Klingebiel, Dietrich Niethammer, Jörn Kühl, Wolfram Ebell, Folker Hanefeld |
Journal | European journal of pediatrics
(Eur J Pediatr)
Vol. 162
Issue 1
Pg. 6-14
(Jan 2003)
ISSN: 0340-6199 [Print] Germany |
PMID | 12486501
(Publication Type: Journal Article)
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Topics |
- Adrenoleukodystrophy
(pathology, therapy)
- Female
- Follow-Up Studies
- Hematopoietic Stem Cell Transplantation
- Humans
- Magnetic Resonance Imaging
- Neurologic Examination
- Neuropsychological Tests
- Treatment Outcome
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