Alirocumab in Pediatric Patients With Heterozygous Familial Hypercholesterolemia: A Randomized Clinical Trial.

ABSTRACT:

Importance

Many pediatric patients with heterozygous familial hypercholesterolemia (HeFH) cannot reach recommended low-density lipoprotein cholesterol (LDL-C) concentrations on statins alone and require adjunct lipid-lowering therapy (LLT); the use of alirocumab in pediatric patients requires evaluation.

Objective

To assess the efficacy of alirocumab in pediatric patients with inadequately controlled HeFH.

Design, setting, and participants

This was a phase 3, randomized clinical trial conducted between May 2018 and August 2022 at 43 centers in 24 countries. Pediatric patients aged 8 to 17 years with HeFH, LDL-C 130 mg/dL or greater, and receiving statins or other LLTs were included. Following consecutive enrollment into dosing cohorts, 25 of 99 patients screened for d

SUBMITTER: Santos RD 

PROVIDER: S-EPMC10845038 | biostudies-literature | 2024 Mar

REPOSITORIES: biostudies-literature