Abstract | BACKGROUND: METHODS: We conducted an open trial in 14 men with adrenomyeloneuropathy, 5 symptomatic heterozygous women, and 5 boys (mean age, 13 years) with preclinical adrenomyeloneuropathy. The patients ate a low-fat diet and received daily doses of glycerol trioleate oil (1.7 g per kilogram of body weight) and glycerol trierucate oil (0.3 g per kilogram). Clinical manifestations, cerebral and spinal cord magnetic resonance imaging (MRI) scans, nerve conduction, and brain-stem auditory and somatosensory evoked potentials were studied prospectively over 18 to 48 months. Plasma levels of very-long-chain fatty acids and the side effects of erucic acid were monitored monthly. RESULTS: By week 10, plasma very-long-chain fatty acid levels declined nearly to normal. Nonetheless, over a mean follow-up of 33 months none of the 14 men with adrenomyeloneuropathy improved. In nine men there was functional deterioration, coincident in four with new cerebral lesions on MRI. In a single patient there was a reduction in cerebellar demyelination, but without clinical improvement. In one of the five asymptomatic boys signs of myelopathy developed. There were no changes in the symptomatic heterozygous women. There was some improvement in peroneal-nerve conduction, but no detectable clinical improvement. Conduction to the parietal cortex (T12-P37 interpeak latency) worsened in both the symptomatic men and the boys with preclinical adrenomyeloneuropathy. There was no change in other somatosensory evoked potentials or in brain-stem auditory evoked potentials. Asymptomatic thrombocytopenia (< 100,000 cells per cubic millimeter) was noted in six patients. CONCLUSIONS:
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Authors | P Aubourg, C Adamsbaum, M C Lavallard-Rousseau, F Rocchiccioli, N Cartier, I Jambaqué, C Jakobezak, A Lemaitre, F Boureau, C Wolf |
Journal | The New England journal of medicine
(N Engl J Med)
Vol. 329
Issue 11
Pg. 745-52
(Sep 09 1993)
ISSN: 0028-4793 [Print] United States |
PMID | 8350883
(Publication Type: Clinical Trial, Journal Article)
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Chemical References |
- Dietary Fats, Unsaturated
- Drug Combinations
- Erucic Acids
- Fatty Acids
- Lorenzo's oil
- Oleic Acids
- Triolein
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Topics |
- Adolescent
- Adrenoleukodystrophy
(blood, diet therapy, physiopathology)
- Adult
- Brain
(pathology, physiopathology)
- Child
- Dietary Fats, Unsaturated
(administration & dosage, therapeutic use)
- Drug Combinations
- Electrophysiology
- Erucic Acids
(administration & dosage, therapeutic use)
- Fatty Acids
(blood)
- Female
- Humans
- Magnetic Resonance Imaging
- Male
- Middle Aged
- Oleic Acids
(administration & dosage, therapeutic use)
- Triolein
(administration & dosage, therapeutic use)
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