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

BMJ. 2008 Nov 11;337:a2423. 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.

Publication types

  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Atorvastatin
  • Cohort Studies
  • Disease-Free Survival
  • Female
  • Heptanoic Acids / administration & dosage*
  • Humans
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors / administration & dosage*
  • Hyperlipoproteinemia Type II / drug therapy*
  • Kaplan-Meier Estimate
  • Male
  • Middle Aged
  • Myocardial Infarction / prevention & control*
  • Pyrroles / administration & dosage*
  • Risk Factors
  • Simvastatin / administration & dosage*
  • Treatment Outcome


  • Heptanoic Acids
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors
  • Pyrroles
  • Atorvastatin
  • Simvastatin