Management of familial hypercholesterolemia in children and young adults: Consensus paper developed by a panel of lipidologists, cardiologists, paediatricians, nutritionists, gastroenterologists, general practitioners and a patient organization

Abstract

a b s t r a c tSince heterozygous familial hypercholesterolemia (HeFH) is a disease that exposes the individual from birth onwards to severe hypercholesterolemia with the development of early cardiovascular disease, a clear consensus on the management of this disease in young patients is necessary. In Belgium, a panel of paediatricians, specialists in (adult) lipid management, general practitioners and representatives of the FH patient organization agreed on the following common recommendations.

Integrated guidance on the care of familial hypercholesterolaemia from the International FH Foundation

“…It is generally agreed that targets for LDL-cholesterol treatment in children need not be as low as adults [8,9,19,20,22,65]. As in adults, good evidence for an absolute or relative target does not exist in children; international guidelines are consequently not uniform in their recommendations [8,9,19,20,22,65].…”

“…As in adults, good evidence for an absolute or relative target does not exist in children; international guidelines are consequently not uniform in their recommendations [8,9,19,20,22,65]. Early cholesterol-lowering treatment can substantially alter the natural history of FH [53,56,[108][109][110][111][112]145].…”

“…Good longterm data on the safety of statins started early in life are therefore required [148,149], emphasizing the need to establish a suitable international registry [150]. The plasma LDL-cholesterol targets for children aged 8 to 10 years should be < 4.0 mmol/L and for those older than 10 years < 3.5 mmol/L [8,19,20,22,65,66,73,145,148]. Boys and girls should be treated to similar targets, but treatment targets could be lowered and therapy intensified in those with a particularly adverse family history of CHD or in the presence of other major cardiovascular risk factors [8,19,43,44,63,65,73].…”

“…Drug treatment will be required in a great majority of patients with heterozygous FH and should be initiated in those with plasma LDL-cholesterol levels (measured on two occasions) above targets after diet management [8,19,20,22,65,73]. Agents tested in clinical trials and approved for use by regulatory agencies should be used [8,19,20,22,65,66,73]; statin drugs are preferred and should be initiated at low doses [146,147].…”

“…Agents tested in clinical trials and approved for use by regulatory agencies should be used [8,19,20,22,65,66,73]; statin drugs are preferred and should be initiated at low doses [146,147]. Statin treatment with approved medications can be initiated at 8-10 years of age and not delayed until 18 years [8,19,20,22,65,66,73].…”