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

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.