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Early statin therapy for familial hypercholesterolaemia slows progression

By Bianca Nogrady
Starting children with familial hypercholesterolaemia on statin therapy could slow the progression of cardiovascular disease, according to a US study published in The New England Journal of Medicine.

Researchers followed 184 patients with familial hypercholesterolaemia – who were originally enrolled in a placebo-controlled trial of the efficacy and safety of pravastatin in children – and 77 of their unaffected siblings for 20 years.

They found that those treated with statins, starting from a mean age of 13 years, showed a 32% mean LDL cholesterol decrease from baseline, with 20% of patients achieving a treatment goal of LDL cholesterol below 100 mg/dL (2.59 mmol/L) at follow up.

The study also saw significant benefits in carotid intima-media thickness. Patients with familial hypercholesterolaemia had greater carotid intima-media thickness at baseline than their unaffected siblings, but by 20 years the mean thickness was similar between the groups (0.555 mm vs 0.551 mm, respectively). However, individuals who achieved the LDL cholesterol target had a lower mean intima-media thickness compared with those who did not (0.532 mm vs 0.56 mm, respectively).

The study also found that the cumulative cardiovascular disease-free survival rate at 39 years was 99% among patients with familial hypercholesterolaemia who were treated with statins from childhood, compared with 74% among their affected parents, after adjusting for sex and smoking status.

Statin treatment also appeared to reduce the risk of death from cardiovascular causes; at 39 years, 100% of the patients with familial hypercholesterolaemia who had been treated with statins since childhood were alive, compared with 93% of their affected parents.

Commenting, preventive cardiologist Professor Ian Hamilton-Craig said the findings reinforced the notion that the earlier familial hypercholesterolaemia was treated with statins, the better.

‘The overall interpretation is that it’s the exposure to LDL cholesterol – not only the length of exposure, but the height of cholesterol levels – that determines the rate of atherosclerosis,’ said Professor Hamilton-Craig, Professor of Preventive Cardiology at Flinders University, Adelaide.

Professor Hamilton-Craig told Medicine Today that the dramatic reductions in the of cardiovascular disease and death among individuals treated with statins from an early age, compared with their affected parents, was consistent with data from adult trials.

‘Starting statin treatment early in children prevented progression of carotid intima thickening and reduced cardiovascular events after 20-year follow up,’ he said.

‘For LDL cholesterol reduction, “longer the better” and “lower the better” are most effective in reducing the progression of atherosclerosis.’
N Engl J Med 2019; 381: 1547-1556.