PMID: 9183487May 1, 1997Paper

Treatment of adrenoleukodystrophy with bone marrow transplantation

Acta Paediatrica
G MalmC Marcus

Abstract

Three children with adrenoleukodystrophy (ALD) underwent allogeneic bone marrow transplantation (BMT) between 1992 and 1993. The first boy had attention deficits, marked neuropsychological deficits and widespread demyelination in the frontal lobes on MRI before transplantation. Four years later he has mentally deteriorated and the demyelination on MRI has progressed. The second boy had no symptoms but had white matter lesions on MRI when diagnosed. He was regularly followed with MRI and neuropsychological investigations until BMT 18 months later. A progress of the lesions was noted on the initial MRI investigations, and 4 months before BMT a worsening of deficits in attention and kinaesthetic praxis could be observed. He rapidly deteriorated after the transplantation and died 18 months later. Both PCR and in situ hybridization confirmed the presence of donor cells in the brain. The third boy had no symptoms but white matter lesions on MRI when diagnosed. The neuropsychological tests remained normal but a slight progress was observed on MRI just before transplantation. This boy is still healthy 3.5 years after BMT. BMT as treatment for ALD has to be considered very early, even if a child without symptoms but signs of demyelinati...Continue Reading

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Citations

Sep 13, 2001·Mental Retardation and Developmental Disabilities Research Reviews·A K Percy, S L Rutledge
Sep 30, 2005·European Journal of Neurology : the Official Journal of the European Federation of Neurological Societies·T HitomiH Shibasaki
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