Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's oil.

Authors

• Hugo W Moser
• Gerald V Raymond
• Shou-En Lu
• Larry R Muenz
• Ann B Moser
• Jiahong Xu
• Richard O Jones
• Daniel J Loes
• Elias R Melhem
• Prachi Dubey
• Lena Bezman
• N Hong Brereton
• Augusto Odone

Publication/Presentation Date

7-1-2005

Abstract

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-microg/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.

Volume

62

Issue

7

First Page

1073

Last Page

1080

ISSN

0003-9942

Published In/Presented At

Moser, H. W., Raymond, G. V., Lu, S. E., Muenz, L. R., Moser, A. B., Xu, J., Jones, R. O., Loes, D. J., Melhem, E. R., Dubey, P., Bezman, L., Brereton, N. H., & Odone, A. (2005). Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's oil. Archives of neurology, 62(7), 1073–1080. https://doi.org/10.1001/archneur.62.7.1073

Disciplines

Business Administration, Management, and Operations | Health and Medical Administration | Management Sciences and Quantitative Methods

PubMedID

16009761

Department(s)

Administration and Leadership

Document Type

Article