From the Guidelines
Atorvastatin is the recommended statin for children aged 10 years and older with suspected heterozygous familial hypercholesterolemia (HeFH), with a typical starting dose of 10 mg once daily, titrated up to a maximum of 20 mg daily based on lipid levels and tolerability. This recommendation is based on the expert panel on integrated guidelines for cardiovascular health and risk reduction in children and adolescents, which strongly recommends the use of statins in children with HeFH and LDL cholesterol levels ≥ 190 mg/dL or ≥ 160 mg/dL with a family history of premature cardiovascular disease and ≥ 2 cardiovascular risk factors 1. The FDA has approved the use of atorvastatin in adolescent boys and postmenarcheal girls aged 10-18 years with HeFH and LDL cholesterol ≥ 190 mg/dL, or ≥ 160 mg/dL with a family history of premature cardiovascular disease and ≥ 2 cardiovascular risk factors 1.
Key Considerations
- The use of statins in children with HeFH should be initiated after lifestyle modifications (diet, exercise) have proven insufficient to adequately lower LDL cholesterol levels.
- Regular monitoring of liver function tests and creatine kinase is recommended, especially at initiation and dose increases.
- Treatment should be supervised by a pediatric lipid specialist or cardiologist experienced in managing familial hypercholesterolemia in children.
- Female adolescents should be counseled about contraception as statins are contraindicated during pregnancy.
Statin Options
- Atorvastatin: approved for use in children aged 10 years and older with HeFH, with a typical starting dose of 10 mg once daily, titrated up to a maximum of 20 mg daily based on lipid levels and tolerability.
- Rosuvastatin: also approved for pediatric use, starting at age 10, with an initial dose of 5-10 mg daily and a maximum of 20 mg daily.
Evidence Summary
The expert panel on integrated guidelines for cardiovascular health and risk reduction in children and adolescents provides strong evidence for the use of statins in children with HeFH, with a focus on atorvastatin as the recommended statin 1. The FDA approval of atorvastatin for use in adolescent boys and postmenarcheal girls aged 10-18 years with HeFH and LDL cholesterol ≥ 190 mg/dL, or ≥ 160 mg/dL with a family history of premature cardiovascular disease and ≥ 2 cardiovascular risk factors, further supports this recommendation 1.
From the FDA Drug Label
Pediatric Patients 10 Years of Age and Older with HeFH In a double-blind, placebo-controlled study, 175 pediatric patients (99 boys and 76 post-menarchal girls) 10 years of age and older (mean age 14 years old) with heterozygous familial hypercholesterolemia (HeFH) were randomized to simvastatin (n=106) or placebo (n=67) for 24 weeks
The simvastatin is approved for use in pediatric patients 10 years of age and older with HeFH. There is no information about approval for use at age 12 in the provided drug label for rosuvastatin. However, simvastatin can be used at age 10 and above, it may be considered for a 12-year-old patient, but the decision should be made by a healthcare professional. 2
From the Research
Statin Treatment for Familial Hypercholesterolemia
- Statins are effective in reducing low-density lipoprotein (LDL) cholesterol levels in children with familial hypercholesterolemia 3, 4.
- The safety of statins in children has been established in the short term, but long-term safety remains unknown 3, 4.
- Statin treatment is recommended to be started in childhood to reduce the risk of cardiovascular disease 3, 4.
Approved Statins for Children
- Atorvastatin, rosuvastatin, and simvastatin have been studied in children with familial hypercholesterolemia 3, 4, 5.
- Rosuvastatin has been shown to be more effective than atorvastatin in reducing LDL cholesterol levels in patients with heterozygous familial hypercholesterolemia 5.
- The choice of statin and dosage should be individualized based on the child's specific needs and response to treatment.
Age of Initiation
- Statin treatment can be initiated in children as young as 6 years old, but the exact age of initiation may vary depending on the individual child's risk factors and disease severity 3, 4.
- In the case of a 12-year-old patient with suspected familial heterozygous hypercholesterolemia, statin treatment may be considered, but the decision should be made on a case-by-case basis, taking into account the child's overall health and response to treatment 6, 7.