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

Hugo W. Moser, Gerald 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

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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-μg/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.

Original languageEnglish (US)
Pages (from-to)1073-1080
Number of pages8
JournalArchives of Neurology
Volume62
Issue number7
DOIs
StatePublished - Jul 1 2005

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Adrenoleukodystrophy
Magnetic Resonance Spectroscopy
Area Under Curve
Fatty Acids
Lorenzo's oil
Oil
Proportional Hazards Models
Disease Progression
Fats
Boys
Confidence Intervals
hexacosanoic acid
Plasma

All Science Journal Classification (ASJC) codes

  • Arts and Humanities (miscellaneous)
  • Clinical Neurology

Cite this

Moser, Hugo W. ; Raymond, Gerald ; Lu, Shou En ; Muenz, Larry R. ; Moser, Ann B. ; Xu, Jiahong ; Jones, Richard O. ; Loes, Daniel J. ; Melhem, Elias R. ; Dubey, Prachi ; Bezman, Lena ; Brereton, N. Hong ; Odone, Augusto. / Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's oil. In: Archives of Neurology. 2005 ; Vol. 62, No. 7. pp. 1073-1080.
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title = "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-μg/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.",
author = "Moser, {Hugo W.} and Gerald Raymond and Lu, {Shou En} and Muenz, {Larry R.} and Moser, {Ann B.} and Jiahong Xu and Jones, {Richard O.} and Loes, {Daniel J.} and Melhem, {Elias R.} and Prachi Dubey and Lena Bezman and Brereton, {N. Hong} and Augusto Odone",
year = "2005",
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Moser, HW, Raymond, G, Lu, SE, Muenz, LR, Moser, AB, Xu, J, Jones, RO, Loes, DJ, Melhem, ER, Dubey, P, Bezman, L, Brereton, NH & Odone, A 2005, 'Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's oil', Archives of Neurology, vol. 62, no. 7, pp. 1073-1080. https://doi.org/10.1001/archneur.62.7.1073

Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's oil. / Moser, Hugo W.; Raymond, Gerald; Lu, Shou En; Muenz, Larry R.; Moser, Ann B.; Xu, Jiahong; Jones, Richard O.; Loes, Daniel J.; Melhem, Elias R.; Dubey, Prachi; Bezman, Lena; Brereton, N. Hong; Odone, Augusto.

In: Archives of Neurology, Vol. 62, No. 7, 01.07.2005, p. 1073-1080.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's oil

AU - Moser, Hugo W.

AU - Raymond, Gerald

AU - Lu, Shou En

AU - Muenz, Larry R.

AU - Moser, Ann B.

AU - Xu, Jiahong

AU - Jones, Richard O.

AU - Loes, Daniel J.

AU - Melhem, Elias R.

AU - Dubey, Prachi

AU - Bezman, Lena

AU - Brereton, N. Hong

AU - Odone, Augusto

PY - 2005/7/1

Y1 - 2005/7/1

N2 - 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-μg/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.

AB - 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-μg/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.

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DO - 10.1001/archneur.62.7.1073

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