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Familial hypercholesterolemia: current treatment options and patient selection for low-density lipoprotein apheresis

Linda C Hemphill1

  • 1Cardiology Department, Massachusetts General Hospital, Yawkey Center, Suite 5800, 55 Fruit Street, Boston, MA 02114, USA. lhemphill@partners.org

Journal of Clinical Lipidology
|December 3, 2010
PubMed

Insights

Familial hypercholesterolemia treatments before statins had severe side effects. Modern statins and LDL apheresis have improved management, but challenges persist in treating this genetic condition.

Area of Science:

  • Cardiology
  • Genetics
  • Metabolic Disorders

Background:

  • Familial hypercholesterolemia (FH) is a genetic disorder causing extremely high cholesterol levels.
  • Severe FH, both heterozygous and homozygous forms, presented limited treatment options pre-statin era, often with significant side effects and morbidity.

Purpose of the Study:

  • To review the historical and current management strategies for severe familial hypercholesterolemia.
  • To highlight the impact of statins and low-density lipoprotein (LDL) apheresis on FH treatment.
  • To identify persistent challenges in managing severe FH.

Main Methods:

  • Literature review of treatment options for familial hypercholesterolemia.
  • Analysis of historical treatment efficacy and side effect profiles.
  • Evaluation of modern therapeutic advancements including statins and apheresis.

Main Results:

  • Pre-statin treatments for severe FH were associated with considerable risks and limited effectiveness.
  • Statins and LDL apheresis represent significant advancements, revolutionizing FH management.
  • Despite progress, challenges in achieving optimal lipid targets and managing complex cases remain.

Conclusions:

  • The management of severe familial hypercholesterolemia has dramatically improved with the introduction of statins and LDL apheresis.
  • Ongoing research and therapeutic innovation are crucial to address remaining challenges in FH treatment.

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