Abstract 18756: Attainment of Low-Density Lipoprotein Cholesterol Treatment Target in Familial Hypercholesterolemia Patients: A Model Exploring Efficacy of Current and Novel Lipid Lowering Therapies
Introduction: Current guidelines recommend low-density lipoprotein cholesterol (LDL-C) levels below 1.8 mmol/L and 2.5 mmol/L for heterozygous familial hypercholesterolemia (FH) patients with and without cardiovascular disease (CVD), respectively. Previous studies report only a small proportion of patients reaching these levels. New therapeutic agents tested in clinical trials have shown to increase these percentages. However, patients included in these trials may not be representative for the general FH population. We set out to quantify the proportion of patients who would reach their target level while using current and novel therapies in an unbiased cohort.
Methods: All adult patients with heterozygous familial hypercholesterolemia identified by the Dutch FH screening program between 1994 and 2014 were included. We quantified the proportion of patients reaching their treatment target in four scenarios: 1) off-treatment (in patients using LLT at time of LDL-C measurement, off-treatment levels were calculated based on type and dose of LLT); 2) after 50% reduction, representing currently available maximally dosed LLT; 3 and 4) additional 40% (CETP inhibition) and 60% reduction (PSCK9 inhibition) respectively on top of maximal LLT. Patients were stratified based on CVD history.
Results: A total of 1,259 adult heterozygous FH patients with and 9,213 without CVD were included. Without treatment, 0.4% of patients with and 2.7% of patients without a medical history of CVD, attained target LDL-C. These proportions were 9.2% and 44.6%, respectively, in scenario 2. In scenario 3, 55.6% of patients with and 92.1 % of patients without CVD reached their treatment target. In scenario 4, this was 93.0% and 99.1%, respectively.
Conclusions: A substantial proportion of heterozygous FH patients will not reach their treatment target using currently available LLT. Addition of PCSK9 or CETP inhibition will, in theory, result in a significant increase in these proportions.
Author Disclosures: M.L. Hartgers: None. J. Besseling: None. G.K. Hovingh: None.
- © 2016 by American Heart Association, Inc.