The effect of atorvastatin on serum lipids and lipoproteins in patients with homozyous familial hypercholesterolemia undergoing LDL-apheresis therapy

Atherosclerosis. 2000 Nov;153(1):89-98. doi: 10.1016/s0021-9150(00)00373-7.


The efficacy of atorvastatin, a new hydroxymethylglutaryl (HMG)-CoA reductase inhibitor, in reducing serum lipid levels, modifying lipoprotein composition, and suppressing cholesterol synthesis was evaluated in patients with homozygous familial hypercholesterolemia (homozygous FH) undergoing LDL-apheresis therapy. Atorvastatin was given in escalating doses (10, 20, and 40 mg/day) to nine patients with homozygous FH. Five of nine patients responded well to atorvastatin; four of these patients were receptor-defective and the remaining one was receptor-negative. The change in LDL-cholesterol in the receptor-defective patients averaged -20.6% compared to the baseline level at the highest dose of atorvastatin. Of five receptor-negative type patients, only one showed good response to atorvastatin therapy with a LDL-cholesterol reduction of 14.9%. Although the other four receptor-negative patients did not show a change in LDL-cholesterol, all of them exhibited a considerable increase in HDL-cholesterol. All patients showed reduced urinary excretion of mevalonic acid, suggesting that atorvastatin decreases LDL-cholesterol by inhibiting cholesterol biosynthesis even where LDL-receptor activity is not present. Atorvastatin also decreased serum triglycerides in both receptor-negative and defective patients, especially in the latter. As cholesterol level rebounds quickly after each apheresis procedure, a combination therapy using atorvastatin and apheresis may increase the efficacy of the apheresis treatment, improving cost-benefit effectiveness by reducing the frequency of the apheresis treatment.

MeSH terms

  • Adolescent
  • Adult
  • Anticholesteremic Agents / adverse effects
  • Anticholesteremic Agents / therapeutic use*
  • Apolipoproteins / blood
  • Atorvastatin
  • Blood Component Removal*
  • Female
  • Heptanoic Acids / adverse effects
  • Heptanoic Acids / therapeutic use*
  • Homozygote
  • Humans
  • Hyperlipoproteinemia Type II / blood*
  • Hyperlipoproteinemia Type II / genetics
  • Hyperlipoproteinemia Type II / therapy*
  • Lipids / blood*
  • Lipoprotein(a) / blood
  • Lipoproteins / blood
  • Lipoproteins, LDL / blood*
  • Lymphocytes / metabolism
  • Male
  • Mevalonic Acid / urine
  • Middle Aged
  • Pyrroles / adverse effects
  • Pyrroles / therapeutic use*
  • Receptors, LDL / metabolism


  • Anticholesteremic Agents
  • Apolipoproteins
  • Heptanoic Acids
  • Lipids
  • Lipoprotein(a)
  • Lipoproteins
  • Lipoproteins, LDL
  • Pyrroles
  • Receptors, LDL
  • Atorvastatin
  • Mevalonic Acid