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Published on: November 10, 2017
Design and baseline data of a pediatric study with rosuvastatin in familial hypercholesterolemia
D Meeike Kusters1, Barbarba A Hutten, Brian W McCrindle
1Department of Vascular Medicine, Academic Medical Center, Amsterdam, The Netherlands; Department of Pediatrics, Academic Medical Center, Amsterdam, The Netherlands.
Insights
Children with familial hypercholesterolemia (FH) show increased carotid intima-media thickness (c-IMT) even at a young age. Early statin therapy initiation is crucial for this high-risk pediatric population.
Area of Science:
- Pediatric Cardiology
- Metabolic Disorders
- Pharmacology
Background:
- Familial hypercholesterolemia (FH) is a genetic condition leading to high LDL cholesterol.
- Current statin therapy guidelines for pediatric FH often fall short of treatment targets.
- Early intervention is critical to mitigate cardiovascular risks in children with FH.
Purpose of the Study:
- To evaluate the safety and efficacy of rosuvastatin in children with heterozygous FH.
- To present the baseline design and findings of the CHARON study.
- To assess rosuvastatin's impact on lipid levels and cardiovascular markers in pediatric FH.
Main Methods:
- An international, 2-year, open-label, titration-to-goal study.
- 198 children (6-18 years) with heterozygous FH and 64 unaffected siblings as controls.
- Rosuvastatin dosage adjusted to achieve lipid goals; primary outcomes: LDL cholesterol change and c-IMT; safety outcomes: growth, sexual maturation, and adverse events.
Main Results:
- At baseline, FH patients (mean age 12.1 years) had elevated LDL cholesterol (6.1 ± 1.3 mmol/L).
- Mean c-IMT was significantly higher in children with FH (0.399 mm) compared to unaffected siblings (0.377 mm) (P = .001).
- These c-IMT differences were evident at a young age, indicating early vascular changes.
Conclusions:
- Children with FH exhibit greater c-IMT than healthy siblings, even in early childhood.
- Baseline findings underscore the importance of early statin initiation in pediatric FH.
- The CHARON study provides insights into rosuvastatin's role in managing pediatric FH.
Background:
Statin therapy is recommended for children with familial hypercholesterolemia (FH), but most children do not reach treatment targets.
Objective:
Here we present the design and results at baseline of the ongoing CHARON study, to evaluate the safety and efficacy of rosuvastatin.
Methods:
This study comprises an international 2-year open label, titration-to-goal study in 198 children with heterozygous FH aged 6 to 18 years, with rosuvastatin in a maximum dose of 10 mg (<10 years of age) or 20 mg (older children). In addition, 64 unaffected siblings were enrolled as controls. The primary efficacy outcome is the change from baseline in low-density lipoprotein cholesterol, and the secondary outcome is the change in carotid intima-media thickness (c-IMT) in patients with FH compared with their siblings. The primary safety outcomes are growth and sexual maturation; secondary outcomes are the change in other lipoprotein levels and the incidence of adverse events, discontinuation rates, and abnormal laboratory values.
Results:
At baseline, mean age of patients with FH was 12.1 ± 3.3 years, 44% were boys, and mean low-density lipoprotein cholesterol levels were 6.1 ± 1.3 mmol/L (235.9 ± 48.7 mg/dL). Mean c-IMT was 0.399 mm (95% CI, 0.392-0.406 mm) in children with FH versus 0.377 (95% CI, 0.366-0.388 mm) in unaffected siblings (P = .001).
Conclusions:
At baseline, as expected according to on previous observations, children with FH proved to have a greater c-IMT than their healthy siblings. These differences had already occurred at a very young age, which emphasizes the importance of considering early statin initiation in this high-risk population.
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