Statin Treatment for Familial Hypercholesterolemia

Study Questions:

What are the national rates of screening, awareness, and treatment with statins among individuals with familial hypercholesterolemia (FH) or severe dyslipidemia?


The investigators used data from the 1999 to 2014 National Health and Nutrition Examination Survey to estimate prevalence rates of self-reported screening, awareness, and statin therapy among US adults (n = 42,471 weighted to represent 212 million US adults) with FH (defined using the Dutch Lipid Clinic criteria) and with severe dyslipidemia (defined as low-density lipoprotein cholesterol levels ≥190 mg/dl). Logistic regression was used to identify sociodemographic and clinical correlates of hypercholesterolemia awareness and statin therapy.


The estimated US prevalence of definite/probable FH was 0.47% (standard error [SE], 0.03%) and of severe dyslipidemia was 6.6% (SE, 0.2%). The frequency of cholesterol screening and awareness was high (>80%) among adults with definite/probable FH or severe dyslipidemia; however, statin use was uniformly low (52.3% [SE, 8.2%] of adults with definite/probable FH and 37.6% [SE, 1.2%] of adults with severe dyslipidemia). Only 30.3% of patients with definite/probable FH on statins were taking a high-intensity statin. The prevalence of statin use in adults with severe dyslipidemia increased over time (from 29.4% to 47.7%), but not faster than trends in the general population (from 5.7% to 17.6%). Older age, health insurance status, having a usual source of care, diabetes mellitus, hypertension, and having a personal history of early atherosclerotic cardiovascular disease were associated with higher statin use.


The authors concluded that despite the high prevalence of cholesterol screening and awareness, only approximately 50% of adults with FH are on statin therapy, with even fewer prescribed a high-intensity statin.


This nationally representative, cross-sectional analysis of the US adult population reports a large disconnect between screening and treatment rates in adults with definite/probable FH and with severe dyslipidemia. Furthermore, although rates of cholesterol screening and awareness approached 90% in adults with definite/probable FH, only about half of these patients were taking a statin at the time of assessment, and only 30% of those on statins were taking a higher-intensity statin. Rates of statin use in adults with FH and severe dyslipidemia appear to be increasing over the past decade, but there remains substantial room for improvement with younger and uninsured adults at greatest risk of being undertreated. We need to better understand how to eliminate the gap between screening and treatment in this high-risk population and improve treatment rates among individuals with limited access to care, thereby reducing cardiovascular morbidity and mortality.

Clinical Topics: Diabetes and Cardiometabolic Disease, Dyslipidemia, Prevention, Homozygous Familial Hypercholesterolemia, Lipid Metabolism, Nonstatins, Novel Agents, Primary Hyperlipidemia, Statins, Hypertension

Keywords: Atherosclerosis, Cholesterol, LDL, Diabetes Mellitus, Dyslipidemias, Hydroxymethylglutaryl-CoA Reductase Inhibitors, Hypercholesterolemia, Hyperlipoproteinemia Type II, Hypertension, Insurance, Health, Metabolic Syndrome X, Primary Prevention

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