Amsterdam UMC study published in the NEJM

Drastic reduction in cardiovascular disease before age⁴⁰ when medical treatment for FH begins in childhood

• A 20-year follow-up study of statin treatment starting in childhood
• Similar arterial wall thickness compared to siblings without FH
• Cardiovascular disease is significantly reduced compared to parents with FH
• Confirmation that early detection and early treatment significantly increase the chances of “aging healthily”

October 17, 2019 – A study by Amsterdam UMC, published this week in The New England Journal of Medicine, shows that the incidence of cardiovascular disease before the age of 40 is drastically reduced when patients with FH (Familial Hypercholesterolemia) begin medical treatment in childhood. The patients began taking statins between the ages of 8 and 18 and were followed for 20 years. Only one of these more than 200 patients underwent angioplasty due to angina pectoris symptoms, while a quarter of their parents had already suffered a heart attack before the age of 40. Read more>>

Previous research—including studies in this same cohort—has shown that untreated FH patients already exhibit functional and morphological changes in the vascular wall during childhood. Partly based on this, it is recommended that these patients be treated with statins starting at age eight. While it was assumed that this would indeed lead to fewer cardiovascular diseases in adulthood, this had never been demonstrated. Over a twenty-year period, the researchers followed more than two hundred children with FH who began taking statins between the ages of eight and eighteen. The children were compared with their siblings (without FH) and with their affected parents (with FH). Because statins were not introduced until 1988, these parents could, on average, start taking statins at the earliest at age 32, whereas their children began taking them at an average age of 14.

No significant side effects occurred during the follow-up period. After 20 years, cholesterol levels in the treated FH patients were found to have decreased significantly from baseline, though they remained higher than those of their unaffected siblings. A striking finding was that the increase in arterial wall thickness was identical between the affected treated and unaffected groups.
Most strikingly, none of the treated children had died from the consequences of cardiovascular disease after more than twenty years of follow-up. Only one patient, who had stopped taking cholesterol-lowering medication after two years of statin treatment, underwent angioplasty at the age of 28 due to angina pectoris. This stands in stark contrast to parents with familial hypercholesterolemia: more than 25% of them experienced a manifestation of cardiovascular disease before the age of forty; and seven percent even died from the consequences of cardiovascular disease!

This study demonstrates the long-term safety and high effectiveness of treating FH patients with statins starting in childhood. This underscores the critical importance of early detection of people with FH—and ideally beginning treatment as early as childhood—in order to completely prevent cardiovascular disease later in life. Unfortunately, there are still 5,000 families with familial hypercholesterolemia in the Netherlands who have not yet been identified…. It is time for action!