Highlights
- •Heterozygous familial hypercholesterolemia can lead to premature cardiovascular disease.
- •To prevent premature cardiovascular disease, statins should be initiated at an early age.
- •CHARON evaluated efficacy and safety of rosuvastin in children aged 6 to 17 years.
- •At 2 years, rosuvastatin 5–20 mg significantly reduced low-density lipoprotein cholesterol up to 45%.
- •Treatment was well tolerated with no adverse effects on growth or sexual maturation.
Objective
Heterozygous familial hypercholesterolemia (HeFH) is an autosomal dominant disorder
leading to premature atherosclerosis. Guidelines recommend initiating statins early
to reduce low-density lipoprotein cholesterol (LDL-C). Studies have evaluated rosuvastatin
in children aged ≥10 years, but its efficacy and safety in younger children is unknown.
Methods
Children with HeFH and fasting LDL-C >4.92 mmol/L (190 mg/dL) or >4.10 mmol/L (>158 mg/dL)
with other cardiovascular risk factors received rosuvastatin 5 mg daily. Based on
LDL-C targets (<2.85 mmol/L [<110 mg/dL]), rosuvastatin could be uptitrated to 10 mg
(aged 6–9 years) or 20 mg (aged 10–17 years). Treatment lasted 2 years. Changes in
lipid values, growth, sexual maturation, and adverse events (AEs) were assessed.
Results
The intention-to-treat analysis included 197 patients. At 24 months, LDL-C was reduced
by 43, 45, and 35% vs baseline in patients aged 6–9, 10–13, and 14–17 years, respectively
(P < .001 for all groups). Most AEs were mild. Intermittent myalgia was reported in
11 (6%) patients and did not lead to discontinuation of rosuvastatin treatment. Serious
AEs were reported by 9 (5%) patients, all considered unrelated to treatment by the
investigators. No clinically important changes in hepatic biochemistry were reported.
Rosuvastatin treatment did not appear to adversely affect height, weight, or sexual
maturation.
Conclusions
In HeFH patients aged 6–17 years, rosuvastatin 5–20 mg over 2 years significantly
reduced LDL-C compared with baseline. Treatment was well tolerated, with no adverse
effects on growth or sexual maturation.
Keywords
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Article info
Publication history
Published online: August 01, 2015
Accepted:
July 26,
2015
Received:
November 11,
2014
Footnotes
M.J.A.M.B. and G.L. contributed equally to the work.
Identification
Copyright
© 2015 National Lipid Association. Published by Elsevier Inc. All rights reserved.
