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… Click to show full abstract
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.
               
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