Heterozygous familial hypercholesterolemia (HeFH) is a condition characterized by elevated plasma concentrations of LDL and LDL-C cholesterol. Evolocumab is a human monoclonal antibody routinely used in patients with lower low-density lipoprotein (LDL) cholesterol levels. This study aims to evaluate the effects of evolocumab in pediatric patients with HeFH.

This double-blind, randomized, placebo-controlled trial included a total of 157 patients aged 10-17 years with HeFH who had received lipid-lowering treatment before. The patients were randomly assigned in a 2:1 ratio to receive evolocumab (420 mg) or placebo. The primary outcome of the study was a percent change in LDL cholesterol levels from the baseline.

Of 157 patients who underwent randomization, 104 received evolocumab, and 53 received a placebo. At week 24, a –44.5% change in LDL cholesterol levels from baseline was recorded in patients in the evolocumab group, compared with –6.2% in the placebo group. The absolute change in the LDL cholesterol levels was –77.5 mg/dL, compared with –9.0 mg/dL in the placebo group. Similar results were observed with all secondary lipid variables.

The research concluded that in pediatric patients with heterozygous familial hypercholesterolemia, treatment with evolocumab produced a superior reduction in LDL cholesterol levels than placebo.

Ref: https://www.nejm.org/doi/full/10.1056/NEJMoa2019910