Immunoablation does not delay the neurologic progression of X-linked adrenoleukodystrophy

J Pediatr. 1997 Sep;131(3):453-5. doi: 10.1016/s0022-3476(97)80076-5.

Abstract

We report the results of a near total myeloablation in preparation for bone marrow transplantation in a boy with minimal symptoms of X-linked adrenoleukodystrophy. Severe cerebral X-linked adrenoleukodystrophy developed in the patient after failure of bone marrow transplantation. This experience suggests that immunotherapy alone is not responsible for the improvement observed in some patients with X-ALD after BMT.

Publication types

  • Case Reports

MeSH terms

  • Bone Marrow Transplantation*
  • Child
  • Disease Progression
  • Genetic Linkage*
  • Humans
  • Magnetic Resonance Imaging
  • Male
  • Peroxisomal Disorders / complications
  • Peroxisomal Disorders / genetics*
  • Peroxisomal Disorders / pathology
  • Peroxisomal Disorders / therapy*
  • Persistent Vegetative State / etiology
  • Transplantation Conditioning*
  • Treatment Failure
  • X Chromosome*