National Lipid Association
National Lipid Association
May 19, 2017
2 min read

Most youth with FH not sufficiently treated for hyperlipidemia

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PHILADELPHIA — Opportunities exist to optimize the treatment of youth with familial hypercholesterolemia to reduce their risk for CV diseases, according to a speaker at the National Lipid Association Scientific Sessions.

“Patients with familial hypercholesterolemia have severe elevations in LDL from birth, leading to increased risk for atherosclerotic [CVD],” Sarah D. De Ferranti, MD, MPH, director of the preventive cardiology program at Boston Children’s Hospital and assistant professor of pediatrics at Harvard Medical School, said during a presentation here. “FH is a common inherited disorder, affecting 1 in every 250 individuals in the United States.”

The new analysis presented a look at the younger population with heterozygous familial hypercholesterolemia (FH), specifically children and adolescents.

Sarah D. De Ferranti

De Ferranti presented results of a cross-sectional analysis of treatment patterns and CV risk factors among 383 children and adolescents enrolled in the CASCADE FH Registry from April 2014 to February 2017 at 18 U.S. lipid clinics. The FH Foundation launched the registry in 2013, with the goal to increase awareness, characterize treatment trends and monitor outcomes associated with FH over time.

Among U.S. youth enrolled in this registry, the mean age at diagnosis was 9.4 years. Half were female and the majority (68%) were white.

“Youth had high LDL, as expected, and nearly two-thirds had additional CV risk factors,” De Ferranti said.

At enrollment, mean LDL was 181 mg/dL. Forty percent of patients had LDL greater than 190 mg/dL. Other baseline lipid measurements were as follows: total cholesterol, 253 mg/dL; HDL, 51 mg/dL; triglycerides, 98.2 mg/dL.

“Youth with FH have additional cardiac risk related to other CV risk factors,” she said. One in 10 children and adolescents with FH had two or more risk factors, above underlying high LDL. The most common additional risk was low HDL, which was present in 37% of the cohort, followed by obesity, which was present in 17% of the cohort.

Half of the children and adolescents were on pharmacotherapy at enrollment, with statins by far the most common (44%). Treatment patterns by age in the registry were also analyzed. Ninety children were aged younger than 10 years and 293 were older than 10 years.

“Statins are recommended for individuals with FH beginning at age 10,” she said.

In this analysis, children aged 10 years and older were twice as likely to be on statin therapy (53% vs. 13%).

Smaller numbers of children and adolescents were using other lipid-lowering therapies including phytosterols, psyllium, ezetimibe (Zetia, Merck), fish oil/omega-3 fatty acids, and bile acid sequestrants.


According to De Ferranti, rates of sufficient LDL lowering were low. At enrollment, only 26% of children and adolescents had LDL below 130 mg/dL or a change in LDL of 50% or greater.

The abstract received the Foundation of the National Lipid Association Hunninghake FH Abstract Award. – by Katie Kalvaitis


De Ferranti SD. Hunninghake FH Abstract Award Winner: Pediatric Familial Hypercholesterolemia: Children and Adolescents Enrolled in the CAscade SCreening for Awareness. Presented at: National Lipid Association Scientific Sessions; May 18-21, 2017; Philadelphia.

Disclosure: de Ferranti reports financial ties with New England Children’s Heart Foundation, PCORI, Pediatric Heart Network and UpToDate.