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Published on: September 15, 2018
20-Year Follow-up of Statins in Children with Familial Hypercholesterolemia
Ilse K Luirink1, Albert Wiegman1, D Meeike Kusters1
1From the Departments of Pediatrics (I.K.L., A.W., D.M.K., J.W.G.), Clinical Epidemiology, Biostatistics, and Bioinformatics (I.K.L., M.H.H., B.A.H.), and Vascular Medicine (I.K.L., J.J.P.K), Amsterdam University Medical Centers, Amsterdam, and Imagelabonline and Cardiovascular, Erichem (E.G.) - both in the Netherlands.
Insights
Early statin therapy for children with familial hypercholesterolemia significantly lowers low-density lipoprotein (LDL) cholesterol and reduces cardiovascular disease risk in adulthood. This 20-year follow-up shows sustained benefits, including slower artery wall thickening.
Area of Science:
- Cardiology
- Genetics
- Pediatrics
Background:
- Familial hypercholesterolemia (FH) causes high LDL cholesterol and early cardiovascular disease.
- Long-term cardiovascular risk data for statin therapy in children with FH is limited.
Purpose of the Study:
- To evaluate the 20-year outcomes of statin therapy initiated in childhood for familial hypercholesterolemia.
- To assess the impact on cardiovascular disease risk and carotid intima-media thickness progression.
Main Methods:
- A 20-year follow-up of 214 children with genetically confirmed FH treated with pravastatin.
- Comparison of cardiovascular events and carotid intima-media thickness with unaffected siblings and affected parents.
- Data collected via questionnaires, blood samples, and intima-media thickness measurements.
Main Results:
- Mean LDL cholesterol decreased by 32% over 20 years, with 20% achieving treatment goals.
- Carotid intima-media thickness progression was similar between FH patients and siblings.
- Cumulative incidence of cardiovascular events and death by age 39 was significantly lower in FH patients compared to affected parents.
Conclusions:
- Childhood initiation of statin therapy in FH patients slows carotid intima-media thickness progression.
- Early statin treatment significantly reduces adult cardiovascular disease risk in individuals with familial hypercholesterolemia.
Background:
Familial hypercholesterolemia is characterized by severely elevated low-density lipoprotein (LDL) cholesterol levels and premature cardiovascular disease. The short-term efficacy of statin therapy in children is well established, but longer follow-up studies evaluating changes in the risk of cardiovascular disease are scarce.
Methods:
We report a 20-year follow-up study of statin therapy in children. A total of 214 patients with familial hypercholesterolemia (genetically confirmed in 98% of the patients), who were previously participants in a placebo-controlled trial evaluating the 2-year efficacy and safety of pravastatin, were invited for follow-up, together with their 95 unaffected siblings. Participants completed a questionnaire, provided blood samples, and underwent measurements of carotid intima-media thickness. The incidence of cardiovascular disease among the patients with familial hypercholesterolemia was compared with that among their 156 affected parents.
Results:
Of the original cohort, 184 of 214 patients with familial hypercholesterolemia (86%) and 77 of 95 siblings (81%) were seen in follow-up; among the 214 patients, data on cardiovascular events and on death from cardiovascular causes were available for 203 (95%) and 214 (100%), respectively. The mean LDL cholesterol level in the patients had decreased from 237.3 to 160.7 mg per deciliter (from 6.13 to 4.16 mmol per liter) - a decrease of 32% from the baseline level; treatment goals (LDL cholesterol <100 mg per deciliter [2.59 mmol per liter]) were achieved in 37 patients (20%). Mean progression of carotid intima-media thickness over the entire follow-up period was 0.0056 mm per year in patients with familial hypercholesterolemia and 0.0057 mm per year in siblings (mean difference adjusted for sex, -0.0001 mm per year; 95% confidence interval, -0.0010 to 0.0008). The cumulative incidence of cardiovascular events and of death from cardiovascular causes at 39 years of age was lower among the patients with familial hypercholesterolemia than among their affected parents (1% vs. 26% and 0% vs. 7%, respectively).
Conclusions:
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. (Funded by the AMC Foundation.).
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