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A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
Published on: September 15, 2018
[Atorvastatin in the treatment of patients with hereditary hypercholesterolemia]
A V Susekov1, T V Balakhonova, O A Pogorelova
1Cardiology Research Complex, 3rd Cherepkovskaya, 15a, 121552 Moscow, Russia.
Insights
Atorvastatin effectively lowers cholesterol in familial hypercholesterolemia patients. The 20 mg dose was well-tolerated, significantly reducing total and LDL cholesterol levels.
Area of Science:
- Cardiology
- Pharmacology
- Metabolic Disorders
Background:
- Familial hypercholesterolemia (FH) is a genetic disorder characterized by high levels of low-density lipoprotein cholesterol (LDL-C).
- Effective lipid-lowering therapy is crucial for managing cardiovascular risk in FH patients.
Purpose of the Study:
- To evaluate the efficacy and tolerability of atorvastatin (20 mg/day) in patients with familial hypercholesterolemia.
- To assess the impact of atorvastatin on lipid profiles and liver enzyme levels.
Main Methods:
- A 3-month study involving 19 patients diagnosed with familial hypercholesterolemia.
- Patients received a daily dose of 20 mg atorvastatin.
- Lipid levels (total cholesterol, LDL-C, triglycerides, HDL-C), atherogenicity index, and liver enzymes (AST, ALT) were monitored.
Main Results:
- Significant reductions observed in total cholesterol (32%), LDL-C (41%), triglycerides (16%), and atherogenicity index (45%).
- A notable increase in high-density lipoprotein cholesterol (HDL-C) of 21% was recorded.
- Atorvastatin was generally well-tolerated, with no significant elevations in AST/ALT; one patient had a mild, asymptomatic ALT increase.
Conclusions:
- Atorvastatin 20 mg/day is an effective treatment for managing lipid levels in familial hypercholesterolemia.
- The medication demonstrated good tolerability, with only 16% of patients discontinuing due to side effects.
- This dosage provides effective lipid control in FH patients.
Abstract:
Efficacy and tolerability of atorvastatin (20 mg/day) were assessed in a 3 month study on 19 patients (5 men, 14 women, mean age 52.3 years) with familial hypercholesterolemia. Average baseline levels of total cholesterol (CH) and low density lipoprotein (LDL) CH were 10.7 and 8.6 mmol/l, respectively. By the end of 3 months levels of CH, LDL CH, triglycerides and atherogeneity index decreased by 32, 41, 16 and 45%, respectively. This was accompanied by 21% increase of high density lipoprotein CH level. There were no cases of AST or ALT activity elevation above 3 upper limits of normal values. However 1 patient had asymptomatic elevation of ALT activity up to 53 U/l which did not cause interruption of therapy. Creatine kinase remained normal throughout the study period. Three patients (16%) stopped taking atorvastatin because of side effects. Thus in patients with familial hypercholesterolemia the dose of atorvastatin 20 mg/day was sufficiently well tolerated and provided effective control of lipid levels.
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