The Knowns and Unknowns of Contemporary Statin Therapy for Familial Hypercholesterolemia
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STATIN DRUGS (R. CESKA, SECTION EDITOR)
The Knowns and Unknowns of Contemporary Statin Therapy for Familial Hypercholesterolemia Jing Pang 1 & Dick C. Chan 1
&
Gerald F. Watts 1,2
# Springer Science+Business Media, LLC, part of Springer Nature 2020
Abstract Purpose of Review Statins are first-line therapy for lowering low-density lipoprotein (LDL) cholesterol in familial hypercholesterolemia (FH), particularly in heterozygous patients. We review advances and new questions on the use of statins in FH. Recent Findings Cumulative evidence from registry data and sub-analyses of clinical trials mandates the value of statin therapy for prevention of atherosclerotic cardiovascular disease (ASCVD) in FH. Statins are safe in children and adolescents with FH, with longer term cardiovascular benefits. The potentially toxic effects of statins in pregnancy need to be considered, but no association has been reported in prospective cohort studies with birth defects. There is no rationale for discontinuation of statins in elderly FH unless indicated by adverse events. FH is undertreated, with > 80% of statin-treated FH patients failing to attain LDL cholesterol treatment targets. This may relate to adherence, tolerability, and genetic differences in statin responsiveness. Statin treatment from childhood may reduce the need for stringent cholesterol targets. Combination of statins with ezetimibe and PCSK9 inhibitors significantly improves the efficacy of treatment. Whether statin use could improve the clinical course of FH patients with COVID-19 and other respiratory infections remains an unsolved issue for future research. Summary Statins are the mainstay for primary and secondary prevention of ASCVD in FH. Sustained long-term optimal statin treatment from an early age can effectively prevent ASCVD over decades of life. Despite their widespread use, statins merit further investigation in FH. Keywords Familial hypercholesterolemia . Cardiovascular disease . Treatment . Statins . Adults . Children
Introduction Familial hypercholesterolemia (FH) is the most common and serious form of inherited high cholesterol that markedly accelerates the onset of atherosclerotic cardiovascular disease (ASCVD), principally coronary artery disease [1, 2]. Patients with FH have a life-long burden of elevated low-density lipoprotein (LDL) that warrants aggressive cholesterol lowering [3]. Statins are frontline for the prevention and treatment of ASCVD in FH [1, 4–9]. We review novel aspects of the use of This article is part of the Topical Collection on Statin Drugs * Gerald F. Watts [email protected] 1
2
statins in the management of FH and explore new avenues for future research.
Review Criteria A search for original articles was performed in the PubMed database using the following key terms: ‘statins’, ‘HMG-CoA reductase inhibitors’, ‘familial hypercholesterolemia’, ‘primary hypercholesterolemia’, ‘LDL-cholesterol’, ‘LDL receptor’, ‘treatment’, and ‘cardiovascular disease’, either alone or in combination. All articles selected were in
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