Pharmacotherapy in familial hypercholesterolemia - Current state and emerging paradigms

Trends Cardiovasc Med. 2023 Apr;33(3):170-179. doi: 10.1016/j.tcm.2021.12.011. Epub 2021 Dec 27.

Abstract

Familial hypercholesterolemia is a highly prevalent but underdiagnosed disease marked by increased risk of cardiovascular morbidity and mortality. Aggressive reduction of LDL-cholesterol is a hallmark of cardiovascular risk mitigation in familial hypercholesterolemia. More recently, we have witnessed an expanded repertoire of pharmacologic agents that directly target LDL-cholesterol and/or reduce heart disease burden. In this state-of-the-art review, we explore the development, clinical efficacy and limitations of existing and potential future therapeutics in familial hypercholesterolemia.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't
  • Research Support, N.I.H., Extramural

MeSH terms

  • Anticholesteremic Agents* / adverse effects
  • Cholesterol, LDL
  • Heart Disease Risk Factors
  • Heart Diseases* / chemically induced
  • Humans
  • Hyperlipoproteinemia Type II* / diagnosis
  • Hyperlipoproteinemia Type II* / drug therapy
  • Hyperlipoproteinemia Type II* / genetics
  • Treatment Outcome

Substances

  • Cholesterol, LDL
  • Anticholesteremic Agents