Immunoablation does not delay the neurologic progression of X-linked adrenoleukodystrophy.
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.
Nowaczyk
M J
MJ
Department of Pediatrics, Hospital for Sick Children, Ontario, Canada.
Saunders
E F
EF
Tein
I
I
Blaser
S I
SI
Clarke
J T
JT
D016026:Bone Marrow Transplantation; D002648:Child; D018450:Disease Progression; D008040:Genetic Linkage; D006801:Humans; D008279:Magnetic Resonance Imaging; D008297:Male; D018901:Peroxisomal Disorders / Q000150:complications / Q000235:genetics* / Q000473:pathology / Q000628:therapy*; D018458:Persistent Vegetative State / Q000209:etiology; D019172:Transplantation Conditioning; D017211:Treatment Failure; D014960:X Chromosome