Clinical score for adrenoleukodystrophy patients candidate for bone marrow transplantation

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Abstract

X-linked adrenoleukodystrophy (ALD) is a progressive genetically determined metabolic disorder that causes severe and progressive nervous system involvement and primary adrenocortical insufficiency. Many different therapeutic approaches have been tried in an attempt to cure this disorder. To date, bone marrow transplantation has been the only one that, in certain selected patients, has given encouraging results, but fewer than 2% of patients undergo this procedure. Candidate patients should have minimal demyelinating lesions as shown by nuclear magnetic imaging of the brain. The aim of the present multidisciplinary study is to evaluate the criteria used in the selection of certain patients with ALD for bone marrow transplantation.

Original languageEnglish
JournalPaediatrics and Child Health
Volume19
Issue numberSUPPL. 2
DOIs
Publication statusPublished - Dec 2009

Keywords

  • bone marrow transplantation
  • clinical score
  • X-linked adrenoleukodystrophy

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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