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Cognitive function with evolocumab in pediatric heterozygous familial hypercholesterolemia

Daniel Gaudet1, Andrea Ruzza2, Ian Bridges3

  • 1The Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC, Canada (Dr Daniel Gaudet).

Journal of Clinical Lipidology
|October 9, 2022
PubMed

Insights

Evolocumab treatment did not negatively affect cognitive function in pediatric patients with familial hypercholesterolemia (FH). Cognitive test scores remained similar between evolocumab and placebo groups over 24 weeks.

Area of Science:

  • Cardiology
  • Neurology
  • Pharmacology

Background:

  • Evolocumab, a PCSK9 inhibitor, is approved for lowering LDL cholesterol in pediatric patients with familial hypercholesterolemia (FH).
  • Cognitive safety of evolocumab is established in adults but not yet in pediatric populations.
  • Familial hypercholesterolemia (FH) is a genetic disorder leading to high LDL cholesterol levels.

Purpose of the Study:

  • To assess the impact of evolocumab on cognitive function in pediatric patients with heterozygous FH.
  • To evaluate the cognitive safety of evolocumab in a pediatric FH cohort.

Main Methods:

  • A 24-week, randomized, double-blind, placebo-controlled study (HAUSER-RCT) was conducted.
  • Cognitive function was assessed using tests for psychomotor function, attention, visual learning, and executive function.
  • Statistical analyses included ANCOVA, Cohen's d, and reliable change indices to compare treatment groups.

Main Results:

  • Cognitive test score changes from baseline were comparable between the evolocumab and placebo groups at 24 weeks.
  • Specific test score differences (95% CI) included: Groton Maze Learning 0.1 (-0.2, 0.4), One-Card Learning -0.1 (-0.5, 0.4), Identification 0.3 (0.0, 0.7), and Detection 0.3 (-0.1, 0.8).
  • Clinically significant cognitive decline occurred less frequently in the evolocumab group.

Conclusions:

  • Evolocumab treatment for 24 weeks demonstrated no adverse effects on cognitive function in pediatric patients with FH.
  • The study supports the cognitive safety profile of evolocumab in this young population.
  • Further long-term studies may be warranted to confirm sustained cognitive safety.
Abstract

Keywords:
ChildCholesterolCognitionCognitive dysfunctionEvolocumabFHFamilial hypercholesterolemiaLDLPCSK9

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