New Insights into Familial Hypercholesterolemia in Children and Adolescents

by Colleen Fleiss on Jan 4 2019 8:22 AM

New Insights into Familial Hypercholesterolemia in Children and Adolescents
New therapeutic approaches, such as mipomersen and PCSK9 inhibitors seem promising for treating familial hypercholesterolemia in children and adolescents, found new study.
Familial hypercholesterolemia is a hereditary genetic disorder predisposing in premature atherosclerosis and cardiovascular complications. Early diagnoses as well as effective treatment strategies in affected children are challenges among experts. Universal screening and cascade screening among families with familial hypercholesterolemia are being controversially discussed.

Treatment approaches for familial hypercholesterolemia in the pediatric population are multidisciplinary and aim to reduce total cardiovascular risk. The most widely recommended and effective pharmacotherapy in the pediatric age group is currently statins. Ezetimibe and bile acid sequestrants are usually used as second-line agents.

Evidence would be expected in the near future by cohort and registry studies.The main gap of evidence remains the lack of longitudinal follow up studies investigating cardiovascular outcomes, side effects, and effectiveness of treatment starting from childhood.