National Lipid Association

National Lipid Association

April 27, 2018
2 min read

Children with FH may benefit from early statin initiation

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LAS VEGAS — Early initiation of statin therapy may reduce CV risk in children with familial hypercholesterolemia, according to a presentation at National Lipid Association Scientific Sessions.

Counseling children on lifestyle modification is the essential first step in caring for children and adolescents with heterozygous FH, Julie St. Pierre, MD, PhD, FAHA, FRCPC, associate professor of pediatrics and lipidology at McGill University in Montreal, said in the presentation. This includes a tobacco-free environment and discouragement of tobacco smoking. The first line of therapy for children and adolescents with heterozygous FH are statins, and adolescent girls should also receive contraception counseling, St. Pierre said.

Within the last 3 years, several papers have been published on proper dosing of statins and when to initiate the therapy in children with familial hypercholesterolemia.

Current recommendations stress the importance of early treatment initiation in homozygous FH, especially since they are fragile patients, St. Pierre said. Once the diagnosis of FH has been supported by strong clinical arguments such as an LDL above 135 mg/dl or a genetic test, statin therapy should be initiated in patients aged 8 to 10 years.

“The treatment target in children is 135 [mg/dL], but the presence of very high LDL levels at first or additional risk factors or high-risk conditions may have to permit us to lower this target to 100 [mg/dL] and also prompt the age of initiation of the therapy before the age of 8 to 10 years old,” St. Pierre said.

Some high-risk conditions and factors include male sex, strong family history of premature CVD or events, high triglycerides, low HDL, overweight or obesity, hypertension, diabetes and elevated lipoprotein(a), she said.

“[These are] many processes that can cause inflammation and accelerate aging of vessels,” St. Pierre said.

The rationale behind this is that the atherosclerotic process takes place earlier in life, which leads to the formation of plaque and the potential acceleration of endothelial dysfunction and events.

The presence of a genetic lipid disorder can accelerate atherosclerosis in patients with heterozygous FH, according to the presentation. If left untreated, children with familial hypercholesterolemia will develop premature plaque and endothelial dysfunction, St. Pierre said, noting heterozygous FH can lead to premature CAD in young adults.

Studies have shown that there were no adverse event differences in children treated with statin therapy vs. placebo. Hepatic and muscle enzymes were also stable in both groups. Bone maturation and puberty staging were unaffected.


In 2015, researchers of the Charon study analyzed data from 197 patients aged 6 years to 17 years with heterozygous FH who were treated with rosuvastatin (Crestor, AstraZeneca) for over 2 years. During that time, LDL was significantly reduced in the treatment arm with no adverse effects on growth or sexual maturation. There were also no clinically important changes in hepatic biochemistry.

Early initiation of statin therapy may be effective in preventing premature CVD and CV mortality, according to the presentation. Patients with heterozygous FH who do not receive treatment can start to have symptoms of CVD as early as 35 years old. Early statin initiation can also slow the increase in carotid intima-media thickness in children with heterozygous FH: In Charon, change in carotid intima-media thickness at 2 years was 0.0054 mm per year in children with heterozygous FH given statins vs. 0.0143 mm per year in their unaffected siblings (P = .002), she said.

“What is very important to point out ... is that doing nothing, the plaque will form very early and will cause dramatic damage to the blood vessel,” St. Pierre said. “Initiating the therapy in childhood will make a huge difference and will keep these [patients] free of CV events.” – by Darlene Dobkowski


St. Pierre J. Familial Hypercholesterolemia in Children. Presented at: National Lipid Association Scientific Sessions; April 26-29, 2018; Las Vegas.

Disclosure: St. Pierre reports she is a primary investigator for an ongoing randomized clinical trial sponsored by Amgen.