Published 11 November 2008, doi:10.1136/bmj.a2423
Cite this as: BMJ 2008;337:a2423

Research

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

¹ Department of Internal Medicine, Erasmus University Medical Centre, PO box 2040, 3000 CA Rotterdam, Netherlands, ² Department of Vascular Medicine, Academic Medical Centre, PO box 22660, 1100 DD Amsterdam, Netherlands, ³ Department of Cardiology, Westfries Gasthuis, PO box 600, 1620 AR Hoorn, Netherlands, ⁴ Department of Cardiology, Oosterschelde Hospital, PO box 106, 4460 BB Goes, Netherlands, ⁵ Department of Epidemiology and Biostatistics, Erasmus University Medical Centre, Rotterdam

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.

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This article has been cited by other articles:

• Mascitelli, L., Pezzetta, F., Goldstein, M. R. (2009). Are Statin Effects Mediated Through, or in Spite of, Their Cholesterol-lowering Action?. ANGIOLOGY 60: 262-263  
• Neil, A., Humphries, S. E (2009). Statins and familial hypercholesterolaemia. BMJ 338: a3041-a3041 [Full text]  

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