title: Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective study.
abstract: 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.
  
  
  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.
  
  
  CONCLUSIONS
  These data suggest that HCT for cerebral ALD in childhood does not prevent the onset of AMN in X-ALD in adulthood.
authors.affiliation: Department of Neurology, Medical Center Alkmaar, Alkmaar, The Netherlands.
authors.forename: Björn M
authors.initials: BM
authors.lastname: van Geel
authors.forename: Bwee Tien
authors.initials: BT
authors.lastname: Poll-The
authors.forename: Aad
authors.initials: A
authors.lastname: Verrips
authors.forename: Jaap-Jan
authors.initials: JJ
authors.lastname: Boelens
authors.forename: Stephan
authors.initials: S
authors.lastname: Kemp
authors.forename: Marc
authors.initials: M
authors.lastname: Engelen
mesh_terms: D000293:Adolescent; D000326:Adrenoleukodystrophy / Q000150:complications / Q000175:diagnosis / Q000601:surgery*; D000328:Adult; D000367:Age Factors; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D012189:Retrospective Studies; D013118:Spinal Cord Diseases / Q000175:diagnosis / Q000209:etiology*; D013997:Time Factors; D016896:Treatment Outcome; D055815:Young Adult