Sammendrag
Abstract
Background and aims: For children with heterozygous familial hypercholesterolaemia (HeFH), European
guidelines recommend consideration of statin therapy by age 8–10 years for those with a low density lipoprotein
cholesterol (LDL-C)>3.5 mmol/l, and dietary and lifestyle advice. Here we compare the characteristics and
lipid levels in HeFH children from Norway, UK, Netherlands, Belgium, Czech Republic, Austria, Portugal and
Greece.
Methods: Fully-anonymized data were analysed at the London centre. Differences in registration and on treatment
characteristics were compared by standard statistical tests.
Results: Data was obtained from 3064 children. The median age at diagnosis differed significantly between countries (range 3–11 years) reflecting differences in diagnostic strategies. Mean (SD) LDL-C at diagnosis was
5.70 (±1.4) mmol/l, with 88% having LDL-C>4.0 mmol/l. The proportion of children older than 10 years at
follow-up who were receiving statins varied significantly (99% in Greece, 56% in UK), as did the proportion
taking Ezetimibe (0% in UK, 78% in Greece). Overall, treatment reduced LDL-C by between 28 and 57%,
however, in those>10 years, 23% of on-treatment children still had LDL-C>3.5 mmol/l and 66% of those not
on a statin had LDL-C>3.5 mmol/l.
Conclusions: The age of HeFH diagnosis in children varies significantly across 8 countries, as does the proportion
of those>10 years being treated with statin and/or ezetimibe. Approximately a quarter of the treated children
and almost three quarters of the untreated children older than 10 years still have LDL-C concentrations over
3.5 mmol/l. These data suggest that many children with FH are not receiving the full potential benefit of early
identification and appropriate lipid-lowering treatment according to recommendations.
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