Management of a patient with a null low-density lipoprotein receptor mutation: a case report

Angiology. 2006;57(6):729-32. doi: 10.1177/0003319706294421.

Abstract

A 13-year-old Greek boy with severe dyslipidemia, large tuberous xanthomas over the knees and elbows, Achilles' tendon xanthomas, and a bilateral corneal arcus was referred to the Lipid Clinic. He had a supravalvular aortic stenosis, 50% to 60% stenosis of both carotid arteries, and normal coronary arteries. Familial hypercholesterolemia was clinically diagnosed. A V408M null low-density lipoprotein receptor (LDLR) mutation was identified in homozygosity. He responded to lipid-lowering drugs by decreasing total cholesterol by 32%, low-density lipoprotein cholesterol by 33%, and triglyceride levels by 30%. Additional treatment with low-density lipoprotein-apheresis further decreased total cholesterol by 52%, low-density lipoprotein cholesterol by 55%, and triglycerides by 43%. Low-density lipoprotein cholesterol levels between apheresis sessions showed a declining pattern. A significant regression of tuberous xanthomas was noted. A suitable combination of lipid-lowering drugs is effective even in this case of homozygosity for a null LDLR mutation. Furthermore, the coadministration of statins, cholestyramine, and ezetimibe during low-density lipoprotein-apheresis tends to counterbalance the postapheresis relapse in low-density lipoprotein cholesterol levels.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Anticholesteremic Agents / therapeutic use
  • Azetidines / therapeutic use
  • Blood Component Removal
  • Cholesterol / blood
  • Cholesterol, LDL / blood
  • Cholestyramine Resin / therapeutic use
  • Ezetimibe
  • Homozygote
  • Humans
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors / therapeutic use
  • Hypercholesterolemia / drug therapy*
  • Hypercholesterolemia / genetics
  • Hypolipidemic Agents / therapeutic use*
  • Male
  • Mutation*
  • Receptors, LDL / genetics*
  • Triglycerides / blood

Substances

  • Anticholesteremic Agents
  • Azetidines
  • Cholesterol, LDL
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors
  • Hypolipidemic Agents
  • Receptors, LDL
  • Triglycerides
  • Cholestyramine Resin
  • Cholesterol
  • Ezetimibe