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Real-World Effectiveness of PCSK9 Inhibitors in Reducing LDL-C in Patients With Familial Hypercholesterolemia in

Marcello Arca1, Simone Celant2, Pier Paolo Olimpieri2

  • 1Department of Translational and Precision Medicine Sapienza University of Rome Rome Italy.

Journal of the American Heart Association
|October 18, 2023
PubMed

Insights

Proprotein convertase subtilisin kexin 9 inhibitors (PCSK9is) demonstrated significant low-density lipoprotein cholesterol (LDL-C) reduction in familial hypercholesterolemia patients in real-world settings. However, achieving target LDL-C goals requires earlier PCSK9i initiation and combination therapies.

Area of Science:

  • Cardiology
  • Pharmacology
  • Genetics

Background:

  • Familial hypercholesterolemia (FH) is a genetic disorder characterized by high low-density lipoprotein cholesterol (LDL-C) levels.
  • Real-world data on the use of proprotein convertase subtilisin kexin 9 inhibitors (PCSK9is) in FH patients are limited.
  • PCSK9is, including alirocumab and evolocumab, offer a novel therapeutic approach for managing hypercholesterolemia.

Purpose of the Study:

  • To evaluate the prescription patterns and long-term efficacy of PCSK9is in Italian patients with familial hypercholesterolemia in a clinical practice setting.
  • To assess the persistence, adherence, and LDL-C reduction achieved with PCSK9is over 24 months.
  • To determine the proportion of patients achieving European Atherosclerosis Society/European Society of Cardiology (EAS/ESC) LDL-C goals.

Main Methods:

  • Analysis of data from the PCSK9i Italian Medicines Agency (AIFA) registry, including patients with heterozygous (HeFH) and homozygous (HoFH) familial hypercholesterolemia.
  • Inclusion of 2484 HeFH and 62 HoFH patients prescribed PCSK9is between February 2017 and December 2021.
  • Evaluation of persistence, adherence, and LDL-C changes over 24 months in a cohort of 1299 FH patients.

Main Results:

  • High persistence and adherence (>85%) to PCSK9i therapy were observed at 6 months.
  • Significant LDL-C reduction was achieved: 58.6% in HeFH (to 79.7 mg/dL) and 57.6% in HoFH (to 95.1 mg/dL) after 24 months.
  • EAS/ESC LDL-C goals were achieved by 43.3% of HeFH patients and 37.5% of HoFH patients.

Conclusions:

  • PCSK9 inhibitors demonstrate efficacy in lowering LDL-C in familial hypercholesterolemia patients in a real-world clinical setting, comparable to controlled trials.
  • A significant proportion of FH patients did not achieve recommended LDL-C goals despite PCSK9i treatment.
  • Achieving optimal LDL-C goals may necessitate earlier initiation of PCSK9is and the use of combination therapies.

Keywords:
LDL cholesterolPCSK9 inhibitorsadherenceheterozygous familial hypercholesterolemiahomozygous familial hypercholesterolemiareal‐world

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