Sensitive analysis of serum 3alpha, 7alpha, 12alpha,24-tetrahydroxy- 5beta-cholestan-26-oic acid diastereomers using gas chromatography-mass spectrometry and its application in peroxisomal D-bifunctional protein deficiency

J Lipid Res. 1998 Dec;39(12):2452-8.

Abstract

The final steps in bile acid biosynthesis take place in peroxisomes and involve oxidative cleavage of the side chain of C27-5beta-cholestanoic acids leading to the formation of the primary bile acids cholic acid and chenodeoxycholic acid. The enoyl-CoA hydratase and beta-hydroxy acyl-CoA dehydrogenase reactions involved in the chain shortening of C27-5beta-cholestanoic acids are catalyzed by the recently identified peroxisomal d-bifunctional protein. Deficiencies of d-bifunctional protein lead, among others, to an accumulation of 3alpha,7alpha,12alpha, 24-tetrahydroxy-5beta-cholest-26-oic acid (varanic acid). The ability to resolve the four C24, C25 diastereomers of varanic acid has, so far, only been carried out on biliary bile acids using p -bromophenacyl derivatives. Here, we describe a sensitive gas chromatography-mass spectrometry (GC/MS) method that enables good separation of the four varanic acid diastereomers by use of 2R-butylester-trimethylsilylether derivatives. This method showed the specific accumulation of (24R,25R)-varanic acid in the serum of a patient with isolated deficiency of the d-3-hydroxy acyl-CoA dehydrogenase part of peroxisomal d-bifunctional protein, whereas this diastereomer was absent in a serum sample from a patient suffering from complete d-bifunctional protein deficiency. In samples from both patients an accumulation of (24S,25S)-varanic acid was observed, most likely due to the action of l-bifunctional protein on Delta24E-THCA-CoA. This GC/MS method is applicable to serum samples, obviating the use of bile fluid, and is a helpful tool in the subclassification of patients with peroxisomal d-bifunctional protein deficiency.

Publication types

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

MeSH terms

  • 17-Hydroxysteroid Dehydrogenases*
  • 3-Hydroxyacyl CoA Dehydrogenases / blood*
  • Cholestanols / blood*
  • Enoyl-CoA Hydratase / blood
  • Gas Chromatography-Mass Spectrometry
  • Humans
  • Hydro-Lyases / blood*
  • Microbodies / metabolism*
  • Multienzyme Complexes / blood*
  • Multienzyme Complexes / deficiency*
  • Peroxisomal Disorders / blood*
  • Peroxisomal Multifunctional Protein-2
  • Predictive Value of Tests
  • Sensitivity and Specificity
  • Stereoisomerism

Substances

  • Cholestanols
  • Multienzyme Complexes
  • 3,7,12,24-tetrahydroxycholestanoic acid
  • 17-Hydroxysteroid Dehydrogenases
  • 3-Hydroxyacyl CoA Dehydrogenases
  • Hydro-Lyases
  • Peroxisomal Multifunctional Protein-2
  • HSD17B4 protein, human
  • Enoyl-CoA Hydratase