Statin therapy initiated during childhood in patients with familial hypercholesterolaemia (FH) slows the rate of carotid intima–media thickening and reduces the risk of cardiovascular events and death. This finding comes from a 20-year follow-up of patients who had previously participated in a 2-year randomized trial of pravastatin therapy.

A total of 214 patients with FH (genetically confirmed in 98%) who had previously participated in the trial and 95 of their unaffected siblings were invited for follow-up. Overall, 79% of the patients with FH reported continued use of lipid-lowering medication.

Among the patients with FH, the mean LDL-cholesterol level had decreased by 32% from 237.3 mg/dl at baseline to 160.7 mg/dl at follow-up. By contrast, in the unaffected siblings, the mean LDL-cholesterol level had increased by 24% from 98.5 mg/dl at baseline to 121.9 mg/dl at follow-up. Mean carotid intima–media thickness was greater in patients with FH than in their unaffected siblings at baseline (0.446 mm versus 0.439 mm). At the 20-year follow-up, the mean carotid intima–media thickness was 0.555 mm in patients with FH and 0.551 mm in their unaffected siblings, indicating a mean rate of thickening of 0.0056 mm per year and 0.0057 mm per year in each group, respectively.

The cumulative incidence of cardiovascular events and death from cardiovascular causes by the age of 39 years was 1% and 0%, respectively, in patients with FH. By contrast, among 156 of their parents with FH (who had not received statin therapy from an early age), the equivalent rates were 26% and 7%, respectively.

These findings support the notion that atherogenesis is the product of both the magnitude and the duration of exposure of the arterial wall to LDL cholesterol and reinforce clinical guideline recommendations to initiate statin therapy by the age of 8–10 years in patients with FH.