A 4-year trial of simvastatin in the treatment of patients with heterozygous familial hypercholesterolemia

A study was conducted to determine the clinical efficacy and tolerability of simvastatin, an inhibitor of 3-hydroxy-3-methylglutaryl coenzyme A reductase in seven patients with heterozygous familial hypercholesterolemia (FH) (defined as primary hypercholesterolemia with tendon xanthoma or primary hypercholesterolemia without tendon xanthomas and at least one first-degree relative with familial hypercholesterolemia), These patients were administrated 10 mg/d of simvastatin for up to 4 years, Simvastatin significantly reduced levels of both total cholesterol and low-density lipoprotein cholesterol during treatment; respective reduction rates were 28% and 34% after 1 month and 32% and 40% after 4 years, Serum high-density lipoprotein cholesterol levels slightly and insignificantly increased, Serum triglyseride levels fell significantly by 24% at month 6, After this study, all xanthomas had reduced in size, No adverse events related to simvastatin were observed. We conclude that long-term simvastatin therapy is clinically useful and well tolerated in patients with FH.