Statin Therapy for Familial Hypercholesterolemia

Initial Statin Selection and Dosing

The American Heart Association recommends high-intensity statin therapy for adults with familial hypercholesterolemia, with the goal of achieving at least a 50% reduction in LDL cholesterol from baseline 1, 2
For pediatric patients with familial hypercholesterolemia (age 10 years and older), start with the lowest dose once daily, usually at bedtime, and consider pravastatin 20 mg daily for children ≤14 years and pravastatin 40 mg daily for children >14 years 4, 5, 6, 7
Rosuvastatin 20-40 mg daily is recommended for adults with familial hypercholesterolemia, as part of high-intensity statin therapy 3, 2

The initial goal is to achieve ≥50% reduction in LDL-C from baseline using high-intensity statin therapy, with target LDL-C levels based on risk, including <2.5 mmol/L (<100 mg/dL) in patients without ASCVD or other major risk factors, <1.8 mmol/L (<70 mg/dL) in patients with imaging evidence of ASCVD or other major risk factors, and <1.4 mmol/L (<55 mg/dL) in patients with clinical ASCVD 1, 2
Titrate statin dose upward if LDL-C goals are not achieved, monitoring for side effects, as recommended by the American College of Cardiology 5, 2

If LDL-C remains ≥100 mg/dL despite maximally tolerated statin therapy, add ezetimibe, as recommended by the American Heart Association 1, 2
Consider adding PCSK9 inhibitors (evolocumab or alirocumab) for adults, or bile acid sequestrants (such as colesevelam), if LDL-C goals are still not achieved with statin plus ezetimibe, as recommended by the American College of Cardiology 1, 2, 8

Before starting statin therapy, measure baseline CK, ALT, AST, glucose, and creatinine, and monitor liver enzymes, muscle enzymes, and glucose as clinically indicated, as recommended by the American Heart Association 5, 2, 8
Instruct patients to report potential adverse effects, especially myopathy (muscle cramps, weakness, asthenia) immediately, and assess relation to physical activity, stop medication, and check CK levels if myopathy occurs, as recommended by the American College of Cardiology 5

For homozygous familial hypercholesterolemia patients, start treatment at diagnosis (ideally by age 2), as recommended by the European Atherosclerosis Society 8
In extremely high-risk familial hypercholesterolemia patients (e.g., after myocardial infarction or with multivessel coronary disease), consider combination of high-potency statin, ezetimibe, and PCSK9-targeted therapy as first-line treatment, as recommended by the American Heart Association 2
Female patients planning pregnancy should be advised about contraception as statins are contraindicated during pregnancy, as recommended by the American College of Obstetricians and Gynecologists 5