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

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.

• 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
• Journal: Archives of neurology (Arch Neurol) Vol. 62 Issue 7 Pg. 1073-80 (Jul 2005) ISSN: 0003-9942 [Print] United States
• PMID: 16009761 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't, Research Support, U.S. Gov't, P.H.S.)

Chemical References

• Biomarkers
• Dietary Fats, Unsaturated
• Drug Combinations
• Erucic Acids
• Fatty Acids
• Lorenzo's oil
• Triolein
• hexacosanoic acid

Topics

• Adrenoleukodystrophy (drug therapy, genetics, pathology)
• Biomarkers (analysis)
• Brain (pathology)
• Child
• Child, Preschool
• Dietary Fats, Unsaturated
• Drug Combinations
• Erucic Acids (therapeutic use)
• Fatty Acids (blood)
• Follow-Up Studies
• Humans
• Magnetic Resonance Imaging
• Male
• Risk Factors
• Treatment Outcome
• Triolein (therapeutic use)