Pediatric dyslipidemias increase the risk of atherosclerosis and clinical cardiovascular disease and are the leading cause of morbidity and mortality. Lifestyle modifications and pharmacotherapies have measurably improved abnormal lipids and reduced cardiovascular events. The review will focus on current standards of care and investigative medications with the potential to improve cardiovascular health in children and adults.

Lifestyle interventions and statins remain cornerstones in the treatment of pediatric hyperlipidemias. Bile acid sequestrants and ezetimibe continue to be used in the pediatric population as well. In recent years, successful clinical trials have approved use of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors in children with familial hypercholesterolemia. Use of angiopoietin-like protein 3 (ANGPTL3) inhibitors is also promising as it causes marked improvement in low-density lipoprotein cholesterol with safe side effect profiles. Additional medications undergoing pediatric clinical trials include inclisiran, bempedoic acid, and lomitapide.

Recent advances in pharmacotherapy, especially for treatment of familial hypercholesterolemia, greatly impact treatment of dyslipidemias in children. Despite the overall progress in the development of these medications, therapies targeted towards treating hypertriglyceridemia have lagged behind. Continuing research for the treatment of pediatric dyslipidemias remains an important endeavor to reduce the risk of atherosclerosis and future cardiovascular events in children.