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Hugo W. Moser, MD; Gerald V. Raymond, MD; Shou-En Lu, PhD; Larry R. Muenz, PhD; Ann B. Moser, BA; Jiahong Xu, MS; Richard O. Jones, PhD; Daniel J. Loes, MD; Elias R. Melhem, MD; Prachi Dubey, MD, MPH; Lena Bezman, MD, MPH; N. Hong Brereton, MS, RD; Augusto Odone

Arch Neurol. 2005;62:1073-1080.

Objectives  To identify asymptomatic boys with X-linked adrenoleukodystrophy who have a normal magnetic resonance image (MRI), and to assess the effect of 4:1 glyceryl trioleate–glyceryl trierucate (Lorenzo’s oil) on disease progression.

Method  Eighty-nine boys (mean ± SD baseline age, 4.7 ± 4.1 years; range, 0.2-15 years) were identified by a plasma very long-chain fatty acids assay used to screen at-risk boys. All were treated with Lorenzo’s oil and moderate fat restriction. Plasma fatty acids and clinical status were followed for 6.9 ± 2.7 years. Changes in plasma hexacosanoic acid levels were assessed by measuring the length-adjusted area under the curve, and a proportional hazards model was used to evaluate association with the development of abnormal MRI results and neurological abnormalities.

Results  Of the 89 boys, 24% developed MRI abnormalities and 11% developed both neurological and MRI abnormalities. Abnormalities occurred only in the 64 patients who were aged 7 years or younger at the time therapy was started. There was significant association between the development of MRI abnormalities and a plasma hexacosanoic acid increase. (For a 0.1-µg/mL increase in the length-adjusted area under the curve for the hexacosanoic acid level, the hazard ratio for incident MRI abnormalities in the whole group was 1.36; P = .01; 95% confidence interval, 1.07-1.72.) Results for patients aged 7 years or younger were similar (P = .04).

Conclusions  In this single-arm study, hexacosanoic acid reduction by Lorenzo’s oil was associated with reduced risk of developing MRI abnormalities. We recommend Lorenzo’s oil therapy in asymptomatic boys with X-linked adrenoleukodystophy who have normal brain MRI results.

Author Affiliations: Kennedy Krieger Institute and Departments of Neurology and Pediatrics, Johns Hopkins University, Baltimore, Md (Drs H. W. Moser, Raymond, A. B. Moser, Jones, Dubey, and Bezman); Division of Biometrics, University of Medicine and Dentistry of New Jersey, Piscataway (Dr Lu); Westat, Rockville, Md (Drs Muenz and Xu); Suburban Radiological Consultants Ltd, Minneapolis, Minn (Dr Loes); Department of Radiology, University of Pennsylvania, Philadelphia (Dr Melhem); General Clinical Research Center, Johns Hopkins School of Medicine, Baltimore (Dr Brereton); and Myelin Project, Dunn Loring, Va (Mr Odone).

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