Plasma very long chain fatty acids in 3,000 peroxisome disease patients and 29,000 controls. 1999

A B Moser, and N Kreiter, and L Bezman, and S Lu, and G V Raymond, and S Naidu, and H W Moser
Department of Neurology, Kennedy Krieger Institute, Baltimore, MD 21205, USA.

The assay of plasma very long chain fatty acids (VLCFAs), developed in our laboratory in 1981, has become the most widely used procedure for the diagnosis of X-linked adrenoleukodystrophy (X-ALD) and other peroxisomal disorders. We present here our 17 years' experience with this assay. Three VLCFA parameters, the level of hexacosanoic acid (C26:0), the ratio of C26:0 to tetracosanoic acid (C24:0), and of C26:0 to docosanoic acid (C22:0), were measured in 1,097 males (hemizygotes) with X-ALD, 1,282 women heterozygous for this disorder, including 379 obligate heterozygotes, 797 patients with other peroxisomal disorders, and 29,600 control subjects. All X-ALD hemizygotes who had not previously received Lorenzo's oil or a diet with a high erucic acid content had increased VLCFA levels, but the application of a discriminant function based on all three measurements is required to avoid the serious consequences of a false-negative result. VLCFA levels are increased at day of birth, thus providing the potential for neonatal mass screening, are identical in the childhood and adult forms, and do not change with age. Eighty-five percent of obligate heterozygotes had abnormally high VLCFA levels, but a normal result does not exclude carrier status. VLCFA levels were increased in all patients homozygous for Zellweger syndrome, neonatal adrenoleukodystrophy, infantile Refsum's disease, and in patients with deficiencies of peroxisomal acyl-coenzyme A oxidase, bifunctional enzyme, and 3-oxoacyl-coenzyme A thiolase. In these patients the degree of VLCFA excess correlated with clinical severity.

UI MeSH Term Description Entries
D007223 Infant A child between 1 and 23 months of age. Infants
D007231 Infant, Newborn An infant during the first 28 days after birth. Neonate,Newborns,Infants, Newborn,Neonates,Newborn,Newborn Infant,Newborn Infants
D008297 Male Males
D008830 Microbodies Electron-dense cytoplasmic particles bounded by a single membrane, such as PEROXISOMES; GLYOXYSOMES; and glycosomes. Glycosomes,Glycosome,Microbody
D008875 Middle Aged An adult aged 45 - 64 years. Middle Age
D010084 Oxidation-Reduction A chemical reaction in which an electron is transferred from one molecule to another. The electron-donating molecule is the reducing agent or reductant; the electron-accepting molecule is the oxidizing agent or oxidant. Reducing and oxidizing agents function as conjugate reductant-oxidant pairs or redox pairs (Lehninger, Principles of Biochemistry, 1982, p471). Redox,Oxidation Reduction
D010641 Phenotype The outward appearance of the individual. It is the product of interactions between genes, and between the GENOTYPE and the environment. Phenotypes
D002648 Child A person 6 to 12 years of age. An individual 2 to 5 years old is CHILD, PRESCHOOL. Children
D002675 Child, Preschool A child between the ages of 2 and 5. Children, Preschool,Preschool Child,Preschool Children
D005188 False Negative Reactions Negative test results in subjects who possess the attribute for which the test is conducted. The labeling of diseased persons as healthy when screening in the detection of disease. (Last, A Dictionary of Epidemiology, 2d ed) False Negative Reaction,Reaction, False Negative,Reactions, False Negative

Related Publications

A B Moser, and N Kreiter, and L Bezman, and S Lu, and G V Raymond, and S Naidu, and H W Moser
January 1992, Advances in experimental medicine and biology,
A B Moser, and N Kreiter, and L Bezman, and S Lu, and G V Raymond, and S Naidu, and H W Moser
July 2022, Progress in lipid research,
A B Moser, and N Kreiter, and L Bezman, and S Lu, and G V Raymond, and S Naidu, and H W Moser
April 2000, Annals of neurology,
A B Moser, and N Kreiter, and L Bezman, and S Lu, and G V Raymond, and S Naidu, and H W Moser
July 1973, Journal of bacteriology,
A B Moser, and N Kreiter, and L Bezman, and S Lu, and G V Raymond, and S Naidu, and H W Moser
October 1981, Neurology,
A B Moser, and N Kreiter, and L Bezman, and S Lu, and G V Raymond, and S Naidu, and H W Moser
February 2022, Current opinion in lipidology,
A B Moser, and N Kreiter, and L Bezman, and S Lu, and G V Raymond, and S Naidu, and H W Moser
February 2022, Biochimica et biophysica acta. Molecular and cell biology of lipids,
A B Moser, and N Kreiter, and L Bezman, and S Lu, and G V Raymond, and S Naidu, and H W Moser
March 1999, European journal of pediatrics,
A B Moser, and N Kreiter, and L Bezman, and S Lu, and G V Raymond, and S Naidu, and H W Moser
June 1994, Journal of neurology, neurosurgery, and psychiatry,
A B Moser, and N Kreiter, and L Bezman, and S Lu, and G V Raymond, and S Naidu, and H W Moser
August 1998, Neurology,
Copied contents to your clipboard!