ISSN 1662-4009 (online)

ey0021.12-14 | Lipid Metabolism | ESPEYB21

12.14. Alirocumab in pediatric patients with heterozygous familial hypercholesterolemia: a randomized clinical trial

RD Santos , A Wiegman , S Caprio , B Cariou , M Averna , Y Poulouin , M Scemama , G Manvelian , G Garon , S Daniels

Brief Summary: This double-blind, randomized trial, showed that 2 dosing regimens of alirocumab, a human monoclonal antibody to proprotein convertase subtilisin kexin type 9 (PCSK9), reduced LDL-C in children as young as 8 years with heterozygous familial hypercholesterolemia inadequately controlled by statins. Efficacy was sustained over 2 years, and both regimens were generally well tolerated.Comment: Heterozygous familial hypercholesterolemia (HeFH) i...