Benefits of early statin therapy in familial hypercholesterolemia



For patients with an inherited form of high blood cholesterol, a 20-year observational study found that the use of statins during childhood slowed atherosclerotic progression, the process by which blockages form in arteries over time, and reduced the risk of cardiovascular disease events in adulthood.


Data was collected from 184 participants with familial hypercholesterolemia, aged between 8 and 18 years. The children who were previously enrolled in a 2-year randomised controlled trial investigating a statin medication were compared to their parents and unaffected siblings. Children were continued on the stain after the 2-year trial had ended.


At follow-up, the participants had an average age of 31 years of which 79% reported they were currently using a lipid-lowering medication and 84% of those had taken the majority of their medication over the past month. Compared with their affected parents for whom statins were only available much later in life, there was a lower cumulative incidence of cardiovascular events at age 39 years (26% vs 1%). None of the participants had died from cardiovascular events compared with 7% of affected parents.


Mean cholesterol levels had decreased 32% from baseline compared to an increase of 24% among their unaffected siblings. 20% had achieved a LDL cholesterol target of less than 100mg/dL. Participants and their siblings had similar carotid artery thickness although those with FH showed greater thickness at baseline.


Why it matters: This long-term study demonstrates the benefits and provides some reassurance over safety concerns of prescribing statins early in life to children with familial hypercholesterolemia to treat this highly preventable cause of cardiovascular disease events.


Source: The New England Journal of Medicine