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Statins for children with familial hypercholesterolemia
Alpo Vuorio1, Jaana Kuoppala, Petri T Kovanen
1Mehilainen Airport Medical Center, Vantaa, Finland.
Insights
Statins effectively lower LDL cholesterol in children with familial hypercholesterolemia. While short-term safety appears good, long-term effects require further investigation in pediatric patients.
Area of Science:
- Cardiovascular Medicine
- Genetics
- Pediatrics
Background:
- Familial hypercholesterolemia (FH) is a common inherited metabolic disorder affecting approximately 1 in 500 individuals worldwide.
- Early diagnosis in children is crucial, often based on elevated low-density lipoprotein cholesterol (LDL-C) levels or DNA analysis, as premature atherosclerosis and cardiovascular complications can occur.
- Traditional treatments like diet and anion exchange resins have limitations in efficacy and tolerability, necessitating alternative therapeutic strategies.
Purpose of the Study:
- To evaluate the effectiveness and safety of statin therapy in pediatric patients diagnosed with familial hypercholesterolemia.
- To synthesize evidence from randomized controlled trials assessing statins versus placebo or diet in children with FH.
Main Methods:
- A systematic search of relevant trials was conducted using the Group's Inborn Errors and Metabolism Trials Register and Medline.
- Eight randomized placebo-controlled trials, including 897 participants up to 18 years of age, were included in the analysis.
- Data on lipid levels, liver enzymes, creatine kinase, and clinical adverse events were extracted and analyzed.
Main Results:
- Statins significantly reduced mean low-density lipoprotein cholesterol (LDL-C) concentrations in children with familial hypercholesterolemia at all measured time points.
- No significant differences were observed in serum aspartate aminotransferase, alanine aminotransferase, or creatine kinase levels between statin and control groups.
- The incidence of myopathy and other clinical adverse events was similar in both treatment and placebo groups, suggesting short-term safety.
Conclusions:
- Statin therapy demonstrates efficacy in lowering LDL-C levels for children with familial hypercholesterolemia.
- While short-term safety appears favorable, the long-term safety profile of statins in this pediatric population remains unknown.
- Careful monitoring by pediatricians is recommended for children on statin therapy, and further long-term randomized controlled trials are warranted.
Background:
Familial hypercholesterolemia is one of the most common inherited metabolic diseases; the average worldwide prevalence of heterozygous familial hypercholesterolemia is about 1 in 500. Diagnosis of familial hypercholesterolemia in children is based on two measurements of low-density lipoprotein cholesterol level above 4.0 mmol/L or a DNA-based analysis. Coronary stenosis has been detected in men with familial hypercholesterolemia as young as 17 years old and in women with familial hypercholesterolemia at 25 years old. Atherosclerosis and its clinical complications occur prematurely, especially in men, thus lifelong hypolipidemic measures, started in childhood, are needed to reduce the risk of cardiovascular diseases. In children with familial hypercholesterolemia children, so far diet has been the main mode of treatment. Anion exchange resins, such as cholestyramine and colestipol, have also been found to be effective but are generally considered unpalatable and therefore poorly tolerated. Since the 1990s statin trials have been carried out among children with familial hypercholesterolemia (aged 7 to 17 years), and statins reduced their serum low-density lipoprotein cholesterol levels by 23% to 40%. The safety of statins among children is not well known even though statins seem to be safe and well-tolerated in adults.
Objectives:
To assess the effectiveness and safety of statins in children with familial hypercholesterolemia.
Search Strategy:
Relevant trials were identified from the Group's Inborn Errors and Metabolism Trials Register and Medline.Date of most recent search: 11 March 2010.
Selection Criteria:
Randomized and controlled clinical trials including participants up to 18 years old comparing a statin to placebo or to diet alone.
Data Collection And Analysis:
Two authors independently assessed studies for inclusion and extracted data.
Main Results:
We found 19 potentially eligible studies of which we included eight randomized placebo-controlled trials (897 participants). Statins reduced the mean low-density lipoprotein cholesterol concentration at all time points. There was no difference between serum aspartate and alanine aminotransferase as well as creatine kinase concentrations at any time-point. The risks of myopathy and clinical adverse events were also similar in both groups. In one study simvastatin was shown to improve flow-mediated dilation of the brachial artery.
Authors' Conclusions:
Statin treatment is an efficient lipid-lowering therapy in children with familial hypercholesterolemia. It seems to be safe in the short term but long-term safety is unknown. Children treated with statins should be carefully followed up by their pediatricians. Large long-term randomized controlled trials are needed to establish the long-term safety of statins.
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