Visual evoked potentials in adrenoleukodystrophy: a trial with glycerol trioleate and Lorenzo oil.
Adrenoleukodystrophy is an X-linked metabolic disorder with very-long-chain fatty acid (VLCFA) accumulation and multifocal nervous system demyelination, often with early involvement of visual pathways. Dietary therapy with glycerol trioleate and glycerol trierucate (Lorenzo oil) diminishes VLCFA levels. In a study of patients with the adrenomyeloneuropathy phenotype of adrenoleukodystrophy, we used pattern-reversal visual evoked potentials to evaluate visual pathways before and after treatment. Of 108 patients tested, all 26 women and 68 of the 82 men had normal potentials at baseline. Seventy patients were retested at 1 year, at which time VLCFA levels were markedly diminished. Of them, the responses in the 10 men who showed abnormalities at baseline remained abnormal; the latencies in 4 men with initially normal responses became abnormal. No patients improved. There were no correlations between disease duration prior to treatment, baseline P100 latencies, VLCFA levels, or the change in P100 latencies and VLCFA levels after dietary treatment for 1 year. Pattern-reversal visual evoked potentials were abnormal in 17% of the men with adrenoleukodystrophy, and there was no evidence that reduction of VLCFA levels improved or retarded visual pathway demyelination.
Kaplan
P W
PW
Department of Neurology, Francis Scott Key Medical Center, Baltimore, MD 21224.
Tusa
R J
RJ
Shankroff
J
J
Heller
J
J
Moser
H W
HW
D000293:Adolescent; D000326:Adrenoleukodystrophy / Q000097:blood / Q000178:diet therapy* / Q000503:physiopathology*; D000328:Adult; D000368:Aged; D004042:Dietary Fats, Unsaturated / Q000627:therapeutic use*; D004338:Drug Combinations; D004883:Erucic Acids / Q000627:therapeutic use*; D005074:Evoked Potentials, Visual; D005227:Fatty Acids / Q000097:blood; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D012737:Sex Factors; D016896:Treatment Outcome; D014304:Triolein / Q000627:therapeutic use*
D004042:Dietary Fats, Unsaturated; D004338:Drug Combinations; D004883:Erucic Acids; D005227:Fatty Acids; C079420:Lorenzo's oil; D014304:Triolein