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Statins in familial hypercholesterolemia: Early initiation leads to better CV outcomes finds NEJM study


Statins in familial hypercholesterolemia: Early initiation leads to better CV outcomes finds NEJM study

Familial hypercholesterolemia is characterized by severely elevated low-density lipoprotein (LDL) cholesterol levels and premature cardiovascular disease.

Early initiation of Statins linked to better CV outcomes in familial hypercholesterolemia, finds a new study. Patients with Familial hypercholesterolemia who initiated statin therapy during childhood had a  reduced risk for CVD later and slower progression of carotid intima-media thickness.The study showed Improved low-density lipoprotein levels and reduced cardiovascular event risk even after 20 years of statin treatment in children with FH. The findings of the study have been published in The New England Journal of Medicine.

Statins are the preferred drug therapy for familial hypercholesterolemia and both the European Atherosclerosis Society consensus panel and American College of Cardiology/American Heart Association guidelines for FH advocate initiating statins at 8 to 10 years of age.

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But the long-term benefit of initiating early statin therapy in children with familial hypercholesterolemia (FH) is unknown.

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The researchers analyzed data from 214 patients with familial hypercholesterolemia (FH) who were assigned statin therapy at age 8 to 18 years in a single-centre, double-blind, placebo-controlled trial that originally evaluated the efficacy and safety of pravastatin at 2 years. Most patients with FH (98%) were genetically confirmed as having the condition. Follow-up in the original study was conducted at a pediatric lipid clinic until patients transitioned to a general practitioner or adult lipid clinic.

The control group consisted of 95 siblings who were unaffected by FH.

Now, researchers have reported outcomes 20 years after statin therapy initiation at ages 8 to 18 years (mean age, 14 years) in 184 patients with FH. The mean age at follow-up was 32 years and the comparison groups were 77 unaffected siblings and 156 affected parents.

At a 20-year follow-up, the researchers found the following results.

  • 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.
The researchers concluded that initiation of statin therapy during childhood in patients with familial hypercholesterolemia slowed the progression of carotid intima–media thickness and reduced the risk of cardiovascular disease in adulthood.The study provides convincing evidence of the value of early statin therapy in children with FH. Therefore family history of FH should prompt attending physician to screen patients for hypercholesterolemia during childhood at around age 8 years and initiate statin therapy as soon as hyperlipidemia is found.

For further reference log on to:

Luirink IK et al. 20-year follow-up of statins in children with familial hypercholesterolemia. N Engl J Med 2019 Oct 17; 381:1547. (https://doi.org/10.1056/NEJMoa1816454)




Source: The New England Journal of Medicine

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