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The Knowns and Unknowns of Contemporary Statin Therapy for Familial Hypercholesterolemia
Jing Pang1, Dick C Chan1, Gerald F Watts2,3
1School of Medicine, Faculty of Health and Medical Sciences, University of Western Australia, Perth, WA, Australia.
Insights
Statins are essential for managing familial hypercholesterolemia (FH), effectively preventing atherosclerotic cardiovascular disease (ASCVD). Early and sustained statin use, often in combination therapy, is crucial for achieving cholesterol targets and long-term cardiovascular health in FH patients.
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Familial hypercholesterolemia (FH) is a genetic disorder characterized by high low-density lipoprotein (LDL) cholesterol levels.
- Statins are the primary pharmacological intervention for managing hypercholesterolemia and preventing atherosclerotic cardiovascular disease (ASCVD).
Purpose of the Study:
- To review current advancements and ongoing questions regarding statin use in patients with familial hypercholesterolemia.
- To evaluate the efficacy and safety of statins in diverse FH populations, including children, adolescents, pregnant women, and the elderly.
Main Methods:
- Review of cumulative evidence from registry data and clinical trial sub-analyses.
- Assessment of safety profiles in pediatric and pregnant populations.
- Analysis of factors contributing to undertreatment and suboptimal LDL cholesterol attainment.
Main Results:
- Statins are proven effective and safe for ASCVD prevention in FH, including in children and adolescents.
- Despite benefits, over 80% of statin-treated FH patients do not reach LDL cholesterol targets, highlighting issues with adherence, tolerability, and response.
- Combination therapies (e.g., statins with ezetimibe, PCSK9 inhibitors) significantly enhance treatment efficacy.
Conclusions:
- Statins are the cornerstone of ASCVD prevention in FH, with early and sustained treatment offering lifelong benefits.
- Addressing undertreatment and optimizing statin regimens, potentially with combination therapies, is critical for improving outcomes in FH.
- Further research is needed to explore statin's role in managing comorbid conditions like COVID-19 in FH patients.
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. 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.
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