Molecular diagnosis of lecithin: cholesterol acyltransferase deficiency in a presymptomatic proband

Clin Chem Lab Med. 1998 Jun;36(7):443-8. doi: 10.1515/CCLM.1998.074.

Abstract

We report the molecular diagnosis of a lecithin : cholesterol acyltransferase deficiency in a 12-year old proband with a high-density lipoprotein deficiency. The increased percentage of free cholesterol in plasma and high-density lipoprotein indicated an inherited lecithin : cholesterol acyltransferase deficiency as the underlying cause. This diagnosis was confirmed by a low plasma lecithin : cholesterol acyltransferase activity and a combination of genetic analyses which demonstrated compound heterozygosity for two mutations in the lecithin : cholesterol acyltransferase gene of the proband. One was a previously unreported 2 bp deletion leading to a stop signal in codon 77 and the other a point mutation causing Arg 135-->Gln transition. To our knowledge, this is the first diagnosis of lecithin : cholesterol acyltransferase deficiency in a pre-symptomatic patient. Whether the proband will develop signs of complete lecithin : cholesterol acyltransferase deficiency or the milder form (Fish Eye Disease) is uncertain, although the former possibility is more likely. The risk of premature atherosclerosis conferred by lecithin : cholesterol acyltransferase deficiency is not well established. The proband will need to be carefully monitored in the future.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Child
  • Cholesterol / blood
  • DNA Mutational Analysis
  • Female
  • Humans
  • Lecithin Cholesterol Acyltransferase Deficiency / blood*
  • Lecithin Cholesterol Acyltransferase Deficiency / genetics
  • Lipids / blood
  • Lipoproteins, HDL / blood
  • Polymerase Chain Reaction
  • Polymorphism, Single-Stranded Conformational

Substances

  • Lipids
  • Lipoproteins, HDL
  • Cholesterol