Statins in Children with Familial Hypercholesterolemia

Published - Written by Ken Wu, MBBS, MRCPCH

Adam is a 15-year-old adolescent diagnosed with familial hypercholesterolemia but is an otherwise healthy teenager with no symptoms. He was started on rosuvastatin therapy when he was 10 years old to reduce the risk of cardiovascular disease. At a routine follow up visit, his pediatrician discusses the transition to adult care within the next few years. Adam is frustrated that he needs to take a tablet every day and admits he often cannot be bothered. He asks whether statins are actually good for his health.

Familial hypercholesterolemia is an autosomal dominant disorder resulting in elevated plasma levels of low-density lipoprotein (LDL). Patients with familial hypercholesterolemia are at higher risk of premature cardiovascular disease and are therefore started on statin therapy from as young as 10 years of age. The lifelong nature of statin therapy presents a major challenge with compliance to treatment, especially in healthy adolescents with no overt symptoms. Patient compliance with statin therapy in adults is also low when used for primary prevention. Information and education about the benefits of treatment can improve compliance to medication in adolescents. Benefits of statin therapy in patients with familial hypercholesterolemia include the reduction of LDL in children and reduced risk of coronary heart disease in adults.

In a 20-year follow-up study recently published in NEJM, starting statin therapy during childhood in patients with familial hypercholesterolemia reduced the risk of cardiovascular disease and slowed the progression of carotid intima-media thickness in adulthood. Adam’s pediatrician can use these findings to reinforce the benefits of statin therapy and help improve compliance with treatment.

The following NEJM Journal Watch summary explains the study and results in more detail.


Long-Term Benefit of Early Statin Therapy for Familial Hypercholesterolemia

Bruder Stapleton, MD reviewing Luirink IK et al. N Engl J Med 2019 Oct 17

Improved low-density lipoprotein levels and reduced cardiovascular event risk were shown after 20 years of statin treatment in children with FH.

The long-term benefit of initiating early statin therapy in children with familial hypercholesterolemia (FH) is unknown. Now, researchers report outcomes 20 years after statin therapy initiation at ages 8 to 18 years (mean age, 14 years) in 184 patients with FH. Mean age at follow-up was 32 years. Comparison groups were 77 unaffected siblings and 156 affected parents.

At 20-year follow-up, results were as follows:

  • Most (79%) of the participants with FH reported still taking statins.
  • Mean LDL cholesterol level was 161 mg/dL (down from 237 mg/dL at study start) compared with 121 mg/dL in unaffected siblings.
  • Mean progression of carotid artery intima-media thickness was not different between participants with FH and their unaffected siblings.
  • Among affected parents, 26% had a cardiovascular event prior to age 40 years, while 99% of the patients who began early statin therapy were free of cardiovascular events after 20 years of therapy.
  • One participant with FH who discontinued statin therapy had an episode of angina pectoris requiring an intervention.
  • Seven percent of affected parents died before age 40 years. No cardiovascular deaths occurred in the study group starting statins as children.

Comment: These data provide convincing evidence of the value of early statin therapy in children with FH. A family history of FH should alert us to screen our patients for hypercholesterolemia at around age 8 years and initiate statin therapy as soon as hyperlipidemia is found.


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 Ken is a 2019-2020 NEJM Editorial Fellow and a paediatrician working in the National Health Service of the United Kingdom. He graduated from Imperial College School of Medicine and is training in general paediatrics in London.