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


METHODS
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.
Moser
Hugo W
HW
Kennedy Krieger Institute, Baltimore, MD 21205, USA. moser@kennedykrieger.org
Raymond
Gerald V
GV
Lu
Shou-En
SE
Muenz
Larry R
LR
Moser
Ann B
AB
Xu
Jiahong
J
Jones
Richard O
RO
Loes
Daniel J
DJ
Melhem
Elias R
ER
Dubey
Prachi
P
Bezman
Lena
L
Brereton
N Hong
NH
Odone
Augusto
A
D000326:Adrenoleukodystrophy / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology*; D015415:Biomarkers / Q000032:analysis; D001921:Brain / Q000473:pathology*; D002648:Child; D002675:Child, Preschool; D004042:Dietary Fats, Unsaturated; D004338:Drug Combinations; D004883:Erucic Acids / Q000627:therapeutic use*; D005227:Fatty Acids / Q000097:blood; D005500:Follow-Up Studies; D006801:Humans; D008279:Magnetic Resonance Imaging; D008297:Male; D012307:Risk Factors; D016896:Treatment Outcome; D014304:Triolein / Q000627:therapeutic use*
D015415:Biomarkers; D004042:Dietary Fats, Unsaturated; D004338:Drug Combinations; D004883:Erucic Acids; D005227:Fatty Acids; C079420:Lorenzo's oil; D014304:Triolein; C017364:hexacosanoic acid