FDA Highlights: Statin therapy shows long-term efficacy in children with inherited cholesterol disorder

by Bruce Sylvester: Ten-year follow-up of children with familial hypercholesterolemia treated with a statin showed an association between statin treatment and normalization of carotid intima-media thickness (IMT), a marker of atherosclerosis, researchers reported in the September 10, 2014 issue of JAMA/The Journal of the American Medical Association.

Familial hypercholesterolemia  is an inherited condition  predisposing a person to premature atherosclerosis and to ensuing cardiovascular disease. Current guidelines suggest initiation of statins in children as young as 8 years. To date, long ­term efficacy and safety data for statin therapy initiated during childhood has not been established.

D. Meeike Kusters, M.D., of the Academic Medical Center, Amsterdam, the Netherlands and colleagues followed for 10 years a cohort of children with familial hypercholesterolemia receiving statin therapy.

The investigators enrolled 214 children heterozygous (with two different forms of a particular gene, one inherited from each parent) for familial hypercholesterolemia, living in the Netherlands, ages 8 to 18 years. The subjects were randomized (in 1997-1999) into a 2-year, placebo-controlled trial of pravastatin.

After the trial, the subjects who received pravastatin were tracked until March 2011, along with 95 unaffected siblings.

At 10 years, all subjects had a thorough physical and blood examination. The investigators acquired evaluable ten-year follow-up data on 194 (91 percent) of the subjects with familial hypercholesterolemia  and 83 (87 percent) of their siblings, all ages 18 to 30 years.

They reported that statin therapy initiated during childhood in subjects with familial hypercholesterolemia was associated with normalization of carotid IMT progression. Notably, earlier initiation of statin treatment was associated with thinner carotid IMT.

No serious adverse events were reported during follow-up.

Notably, at 10 years, low-density lipoprotein levels in familial hypercholesterolemia subjects did not meet current treatment standards and carotid IMT was thicker than in unaffected siblings. “More robust lipid-lowering therapy or earlier initiation of statins may be required to completely restore arterial wall morphology and avert cardiovascular events later in life in this high-risk population,” the authors concluded.