Santos RD, Ruzza A, Hovingh GK, et al. - Because evolocumab, a fully human monoclonal antibody directed against proprotein convertase subtilisin–kexin type 9, is widely used in adult patients to lower low-density lipoprotein (LDL) cholesterol levels, researchers tested the safety and effectiveness of evolocumab in pediatric patients with heterozygous familial hypercholesterolemia. For this 24-week, randomized, double-blind, placebo-controlled trial, patients, aged 10 to 17 years, who had received stable lipid-lowering treatment for at least 4 weeks prior to screening and who had an LDL cholesterol level of 130 mg per deciliter (3.4 mmol per liter) or more and a triglyceride level of 400 mg per deciliter (4.5 mmol per liter) or less were assigned randomly in a 2:1 ratio to receive monthly subcutaneous injections of evolocumab (420 mg) or placebo. In total, 157 patients underwent randomization and received evolocumab (104 patients) or placebo (53 patients). Evolocumab reduced the LDL cholesterol level and other lipid variables in this trial involving pediatric patients with familial hypercholesterolemia. In the evolocumab and placebo groups, the incidence of adverse events that occurred during the treatment period was comparable.