LAUSR

In the HAUSER randomized, placebo­ controlled clinical trial, paediatric patients (aged 10–17 years) with heterozygous familial hypercholesterolaemia treated with evolocumab (a fully human monoclonal antibody against PCSK9) for 24 weeks had a 44.5% mean reduction in LDL­ cholesterol levels compared with a 6.2% mean reduction in those who received placebo (between­ group difference 38.3 percentage points, P < 0.001). The absolute reduction in LDL­ cholesterol level between baseline and week 24 was 77.5 mg/dl in the evolocumab group compared with 9.0 mg/dl in the placebo group (between­ group difference 68.6 mg/dl, P < 0.001). The incidence of adverse events during the treatment period was similar in both groups.