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-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.
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Neonatal screening for ALD was initially proposed by Moser and colleagues in 2004-2005.
“Neonatal screening for ALD was initially proposed by Moser and colleagues in 2004-2005 (50).”
From X-linked adrenoleukodystrophy and primary adrenal insufficiency (2023) · cites this paper as [50]
Cites
Moser, H. W., Mahmood, A., & Raymond, G. V. (2007) · Nature Clinical Practice Neurology · doi:10.1038/ncpneuro0421 · Cited by 663 · see citing works
Self-citationA Two-Year Trial of Oleic and Erucic Acids (“Lorenzo's Oil”) as Treatment for Adrenomyeloneuropathy
Aubourg, P., Adamsbaum, C., Lavallard-Rousseau, M.-C., Rocchiccioli, F., Cartier, N., Jambaque, I., Jakobezak, C., Lemaitre, A., Boureau, F., Wolf, C., et al. (1993) · New England Journal of Medicine · doi:10.1056/nejm199309093291101 · Cited by 242 · see citing works
Moser, H. W., Loes, D. J., Melhem, E. R., Raymond, G. V., Bezman, L., Cox, C. S., & Lu, S.-E. (2000) · Neuropediatrics · doi:10.1055/s-2000-9236 · Cited by 210 · see citing works
Self-citationDubey, P., Raymond, G. V., Moser, A. B., Kharkar, S., Bezman, L., & Moser, H. W. (2005) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2004.10.067 · Cited by 145 · see citing works
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Moser, H. W., Mahmood, A., & Raymond, G. V. (2007) · Nature Clinical Practice Neurology · doi:10.1038/ncpneuro0421 · Cited by 663 · see citing works
Self-citationAdrenoleukodystrophy—Neuroendocrine pathogenesis and redefinition of natural history
Kemp, S., Huffnagel, I. C., Linthorst, G. E., Wanders, R. J., & Engelen, M. (2016) · Nature Reviews Endocrinology · doi:10.1038/nrendo.2016.90 · Cited by 275 · see citing works
Adrenoleukodystrophy: New Approaches to a Neurodegenerative Disease
Moser, H. W., Raymond, G. V., & Dubey, P. (2005) · JAMA · doi:10.1001/jama.294.24.3131 · Cited by 142 · see citing works
Self-citationTreatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy
Gupta, A. O., Raymond, G., Pierpont, E. I., Kemp, S., McIvor, R. S., Rayannavar, A., Miller, B., Lund, T. C., & Orchard, P. J. (2022) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2022.2124857 · Cited by 67 · see citing works
“Reproduced with permission from [41], © 2005 American Medical Association.”
BackgroundMethodSelf-citationABCD1 dysfunction alters white matter microvascular perfusion
Lauer, A., Da, X., Hansen, M. B., Boulouis, G., Ou, Y., Cai, X., Liberato Celso Pedrotti, A., Kalpathy-Cramer, J., Caruso, P., Hayden, D. L., et al. (2017) · Brain · doi:10.1093/brain/awx262 · Cited by 48 · see citing works
X-linked adrenoleukodystrophy and primary adrenal insufficiency
Cappa, M., Todisco, T., & Bizzarri, C. (2023) · Frontiers in Endocrinology · doi:10.3389/fendo.2023.1309053 · Cited by 28 · see citing works
- Authors
- Moser, H. W., Raymond, G. V., Lu, S.-E., Muenz, L. R., Moser, A. B., Xu, J., Jones, R. O., Loes, D. J., Melhem, E. R., Dubey, P., Bezman, L., Brereton, N. H., & Odone, A.
- Affiliations
- Kennedy Krieger Institute (US)
- Rutgers, The State University of New Jersey (US)
- Westat (United States) (US)
- University of Pennsylvania (US)
- Johns Hopkins University (US)
- Myelin Project (US)
- ORCID
- Venue
- Archives of Neurology
- Publisher
- American Medical Association (AMA)
- Published
- 2005-07-01
- Volume
- 62
- Issue
- 7
- Pages
- 1073
- ISSN
- 0003-9942 (ISSN-L)
- OpenAlex
- W2109846359
- Cited by
- 239
- References
- 4
- Citations by year
- 2026: 3 · 2025: 7 · 2024: 8 · 2023: 7 · 2022: 6 · 2021: 8 · 2020: 8 · 2019: 10 · 2018: 9 · 2017: 15 · 2016: 14 · 2015: 7 · 2014: 12 · 2013: 13 · 2012: 7
- FWCI
- 13.42
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Archives of Neurology (publishedVersion)
- PubMed (publishedVersion)
- Funders
- U.S. Public Health Service (685-008)
- National Center for Research Resources (M01 RR-00052, M01RR000052)
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (R01HD039276, HD-10981, HD39276, P01HD010981)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Life Sciences › Neuroscience › Neurology › Neurological diseases and metabolism
- Keywords
- X-linked adrenoleukodystrophy, Lorenzo's oil, disease progression, MRI abnormalities, hexacosanoic acid, very long chain fatty acids, plasma fatty acids, dietary fat restriction, neurological abnormalities, treatment follow-up
- MeSH terms
- Adrenoleukodystrophy, Brain, Child, Child, Preschool, Dietary Fats, Unsaturated, Drug Combinations, Erucic Acids, Fatty Acids, Follow-Up Studies, Humans, Magnetic Resonance Imaging, Male, Risk Factors, Triolein, Biomarkers, Treatment Outcome
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