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

Arch Neurol. 2005 Jul;62(7):1073-80. doi: 10.1001/archneur.62.7.1073.


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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adrenoleukodystrophy / drug therapy*
  • Adrenoleukodystrophy / genetics
  • Adrenoleukodystrophy / 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*


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