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Blood, 1 August 2004, Vol. 104, No. 3, pp. 881-888.
Prepublished online as a Blood First Edition Paper on April 8, 2004; DOI 10.1182/blood-2003-10-3402.
 Previous Article | Next Article 
Submitted October 16, 2003
Accepted March 23, 2004
Cerebral X-linked adrenoleukodystrophy: The international hematopoietic cell transplantation experience from 1982 to 1999
Charles Peters*, Lawrence R Charnas, Ye Tan, Richard S Ziegler, Elsa G Shapiro, Todd DeFor, Satkiran S Grewal, Paul J Orchard, Susan L Abel, Anne I Goldman, Norma K Ramsay, Kathryn E Dusenbery, Daniel J Loes, Lawrence A Lockman, Shunichi Kato, Patrick R Aubourg, Hugo W Moser, and William Krivit
Pediatrics, University of Minnesota, Minneapolis, MN, USA
Biostatistics, University of Minnesota, Minneapolis, MN, USA
Therapeutic Radiology, University of Minnesota, Minneapolis, MN, USA
Suburban Radiologic Consultants, Ltd., Minneapolis, MN, USA
Tokai University, Isehara, Japan
Hopital Saint-Vincent de Paul, INSERM, Paris, France
Kennedy Krieger Institute, Baltimore, MD, USA
* Corresponding author; email: peter392{at}umn.edu.
Cerebral X-linked adrenoleukodystrophy (X-ALD) is a disorder of very-long-chain fatty acid metabolism, adrenal insufficiency, and cerebral demyelination. Death occurs within 2 to 5 years of clinical onset without hematopoietic cell transplantation (HCT). One hundred twenty-six boys with X-ALD received HCT from 1982 to 1999. Survival, engraftment, and acute graft-versus-host disease were studied. Degree of disability associated with neurologic and neuropsychological function and cerebral demyelination were evaluated before and after HCT. Complete data were available and analyzed for 94 boys with cerebral X-ALD. The estimated 5 and 8-year survival was 56%. The leading cause of death was disease progression. Donor-derived engraftment occurred in 86% of patients. Demyelination involved parietal-occipital lobes in 90% leading to visual and auditory processing deficits in many boys. Overall 5-year survival of 92% in patients with zero or one neurologic deficits and MRI severity score < 9 before HCT was superior to survival for all others (45%; P<.01). Baseline neurologic and neuropsychologic function, degree of disability and neuroradiologic status predicted outcomes following HCT. In this first comprehensive report of the international HCT experience for X-ALD, we conclude that boys with early stage disease benefit from HCT while boys with advanced disease may be candidates for experimental therapies.
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