Efficacy of statins in familial hypercholesterolaemia: a long term cohort study

• Published in: BMJ Vol. 337; no. 7688; pp. 223 - 226
• Main Authors: Versmissen, Jorie, Oosterveer, Daniëlla M, Yazdanpanah, Mojgan, Defesche, Joep C, Basart, Dick C G, Liem, Anho H, Heeringa, Jan, Witteman, Jacqueline C, Lansberg, Peter J, Kastelein, John J P, Sijbrands, Eric J G
• Format: Journal Article
• Language: English
• Published: England British Medical Journal Publishing Group 11.11.2008; British Medical Association; BMJ Publishing Group LTD; BMJ Publishing Group Ltd
• Subjects: Adult; Angina pectoris; Angioplasty; Atherosclerosis; Atorvastatin Calcium; Cardiovascular disease; Cardiovascular diseases; Children; Cholesterol; Cholesterols; Cohort Studies; Coronary artery disease; Disease risk; Disease-Free Survival; Female; Heart attacks; Heptanoic Acids - administration & dosage; Humans; Hydroxymethylglutaryl-CoA Reductase Inhibitors - administration & dosage; Hyperlipoproteinemia Type II - drug therapy; Kaplan-Meier Estimate; Lipids; Low density lipoprotein; Male; Medical prognosis; Middle Aged; Mortality; Myocardial infarction; Myocardial Infarction - prevention & control; Older adults; Pyrroles - administration & dosage; Risk Factors; Simvastatin - administration & dosage; Statins; Treatment Outcome
• ISSN: 0959-8138; 1756-1833; 1468-5833; 1756-1833
• DOI: 10.1136/bmj.a2423

Objective To determine the efficacy of statin treatment on risk of coronary heart disease in patients with familial hypercholesterolaemia.Design Cohort study with a mean follow-up of 8.5 years.Setting 27 outpatient lipid clinics.Subjects 2146 patients with familial hypercholesterolaemia without prevalent coronary heart disease before 1 January 1990.Main outcome measures Risk of coronary heart disease in treated and “untreated” (delay in starting statin treatment) patients compared with a Cox regression model in which statin use was a time dependent variable.Results In January 1990, 413 (21%) of the patients had started statin treatment, and during follow-up another 1294 patients (66%) started after a mean delay of 4.3 years. Most patients received simvastatin (n=1167, 33 mg daily) or atorvastatin (n=211, 49 mg daily). We observed an overall risk reduction of 76% (hazard ratio 0.24 (95% confidence interval 0.18 to 0.30), P<0.001). In fact, the risk of myocardial infarction in these statin treated patients was not significantly greater than that in an age-matched sample from the general population (hazard ration 1.44 (0.80 to 2.60), P=0.23).Conclusion Lower statin doses than those currently advised reduced the risk of coronary heart disease to a greater extent than anticipated in patients with familial hypercholesterolaemia. With statin treatment, such patients no longer have a risk of myocardial infarction significantly different from that of the general population.