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Hugo W. Moser, MD; Asif Mahmood, MD, MPH

Arch Neurol. 2007;64(5):631-632.

	Since this article does not have an abstract, we have provided the first 150 words of the full text and any section headings.

Hematopoietic stem cell transplantation (HSCT) can arrest the progression of the childhood or adolescent cerebral forms of X-linked adrenoleukodystrophy (X-ALD), provided that it is administered when the disorder is still in its early stage. This was first demonstrated by Aubourg et al¹ in 1990. Shapiro et al² demonstrated that the stabilization can persist for 5 to 10 years. The most comprehensive follow-up study is that of Peters et al³ who reported on the outcome of 126 patients with childhood cerebral X-ALD who had received HSCT between 1982 and 1999. They showed that the disease stage at time of HSCT has a striking effect on outcome. Five-year survival was 92% in mildly involved patients (0 or 1 neurological deficits and a score of <9 on the 34-point magnetic resonance imaging severity score designed by Loes et al).⁴ In patients who underwent transplantation at . . . [Full Text of this Article]

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