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Statins Could Lower Future CVD Risk in Children with Familial Hypercholesterolemia

October 17, 2019

Beginning statin therapy in childhood could help to reduce the risk of cardiovascular disease later in life among children with familial hypercholesterolemia (FH), according to the results of a recent study.

Although the short-term efficacy of statins has been established in children, there is little information available on these patients’ cardiovascular outcomes later in life, according to the researchers.

They conducted a 20-year follow-up study including 214 children with FH who had previously taken part in a placebo-controlled trial evaluating the safety and efficacy of pravastatin. Also included in the study were 95 unaffected siblings of the participants.

Blood samples and questionnaire answers were collected from the participants, and carotid intima-media thickness was measured. Incidence of cardiovascular disease was compared among the children with FH and 156 of their affected parents.

Overall, 184 of the 214 patients with FH and 77 of the 95 siblings were evaluated at follow-up, with data on cardiovascular outcomes available for 203 of the 214. Mortality data was available for all 214 participants with FH.

Mean LDL cholesterol level decreased 32% from 237.3 to 160.7 mg per deciliter and treatment goals (LDL <100 mg per deciliter [2.59 mmol per liter]) were achieved in 37 of the participants. Mean progression of carotid intima-media thickness was 0.0056 mm per year in the patients with FH and 0.0057 mm per year in their unaffected siblings.

At 39 years of age, the cumulative incidence of cardiovascular events and mortality was lower in the participants with FH than among their affected parents (1% vs 26% and 0% vs 7%, respectively).

“In this study, initiation of statin therapy during childhood in patients with familial hypercholesterolemia slowed the progression of carotid intima–media thickness and reduced the risk of cardiovascular disease in adulthood,” the researchers concluded.

—Michael Potts


Luirink IK, Wiegman A, Kusters M, et al. 20-year follow-up of statins in children with familial hypercholesterolemia [published online October 17, 2019]. NEJM. doi: 10.1056/NEJMoa1816454.

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