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) oratorvastatin (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 ratio 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. [ABSTRACT FROM AUTHOR]