Volume 23, Issue 3 pp. 273-277
Article

Therapy of X-linked adrenoleukodystrophy: Prognosis based upon age and MRI abnormality and plans for placebo-controlled trials

H. W. Moser

H. W. Moser

Kennedy Krieger Institute and the Departments of Neurology and Pediatrics, Johns Hopkins University and the Department of Biostatistics, School of Public Health, 707 North Broadway, Baltimore, MD, 21205 USA

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L. Bezman

L. Bezman

Kennedy Krieger Institute and the Departments of Neurology and Pediatrics, Johns Hopkins University and the Department of Biostatistics, School of Public Health, 707 North Broadway, Baltimore, MD, 21205 USA

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S. E. Lu

S. E. Lu

Kennedy Krieger Institute and the Departments of Neurology and Pediatrics, Johns Hopkins University and the Department of Biostatistics, School of Public Health, 707 North Broadway, Baltimore, MD, 21205 USA

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G. V. Raymond

G. V. Raymond

Kennedy Krieger Institute and the Departments of Neurology and Pediatrics, Johns Hopkins University and the Department of Biostatistics, School of Public Health, 707 North Broadway, Baltimore, MD, 21205 USA

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First published: 01 May 2000
Citations: 14

Abstract

Evaluation of the therapy of X-linked adrenoleukodystrophy (X-ALD) is hampered by its rarity and by the striking and unpredictable variation in phenotypic expression. We present two approaches that may facilitate therapy evaluation. (1) We have analysed data on 377 X-ALD patients who have been followed at the Kennedy Krieger Institute for a mean period of 38 months and have subdivided them into 18 subgroups on the basis of age and the degree of abnormality in brain magnetic resonance imaging (MRI) as assessed by the Loes score (Am. J. Neuroradiol 1994; 15: 1761). We find that grouping on the basis of age and MRI score provides information that is of significant prognostic value. (2) We present plans for the development of a placebo-controlled multicentre international study that will have sufficient biostatistical power to provide objective evaluation of new therapeutic interventions.

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