Early-onset plasmapheresis and LDL-apheresis provide better disease control for pediatric homozygous familial hypercholesterolemia than HMG-CoA reductase inhibitors and ameliorate atherosclerosis

Transfus Apher Sci. 2013 Oct;49(2):268-77. doi: 10.1016/j.transci.2013.05.001. Epub 2013 Jun 21.

Abstract

Plasmapheresis (PA) and low-density lipoprotein apheresis (LDL-A) were assessed in five children with homozygous familial hypercholesterolemia (HFH) previously receiving statins. LDL-A required smaller extracorporeal blood volumes. Mean HDL-cholesterol reduction post-procedure was 32% and 60% with LDL-A and PA, respectively. Non-HDL-C reduction was 64% and 69%, respectively. Pre-procedural LDL-C decreased significantly with weekly versus biweekly LDL-A. Carotid intimal media thickness (IMT) studies demonstrated disappearance of atheromatous lesions and normal ITM in 4/5 patients. Echocardiography revealed normal aortic valves, coronary orifices and supra-valvular area in all patients. Apheresis is effective in pediatric HFH. It may be started in patients weighing 14 kg.

Keywords: Children; Familial hypercholesterolemia; LDL-apheresis; Plasmapheresis.

Publication types

  • Clinical Trial

MeSH terms

  • Atherosclerosis / blood
  • Atherosclerosis / physiopathology
  • Atherosclerosis / prevention & control*
  • Carotid Intima-Media Thickness
  • Child
  • Child, Preschool
  • Cholesterol, HDL / blood
  • Female
  • Humans
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors / therapeutic use
  • Hyperlipoproteinemia Type II / blood
  • Hyperlipoproteinemia Type II / complications
  • Hyperlipoproteinemia Type II / physiopathology
  • Hyperlipoproteinemia Type II / therapy*
  • Lipoproteins, LDL*
  • Male
  • Plasmapheresis*

Substances

  • Cholesterol, HDL
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors
  • Lipoproteins, LDL