20-Year Follow-up of Statins in Children with Familial Hypercholesterolemia

Comparison of the characteristics at diagnosis and treatment of children with heterozygous familial hypercholesterolaemia (FH) from eight European countries

“…Since ultrasound studies have demonstrated significant carotid intima-media thickening in non-treated FH children of this age compared to their non-FH siblings [34][35][36], and clinical trials have shown that statin treatment can reverse this [19,37,38], considering initiation of statin therapy by the age of 8-10 years is a recommendation in most recent guidelines [9][10][11][12][13][14]. While, for ethical and practical reasons, there are no long term randomised-placebo controlled trials to examine the benefit of statin initiation at this age and LDL-C level, observational studies over at least 20 years support the reduction of CVD risk associated with this approach [17]. While the proportion of children over the age of 10 years being treated with ezetimibe varies widely across Europe, the vast majority of those taking this medication do achieve the 3.5 mmol/l LDL-C target.…”

“…obesity or level of Lp(a)] [16]. Although follow-up of children with FH who were started on a statin by the age of 10 years supports the potential CHD benefit [11,17], the age at which statin use should be started, or its intensity to best prevent the onset of adult premature CHD has not been rigorously established, since there are no long-term randomized controlled outcome trials for ethical and practical reasons. There is, however, considerable short term randomized and observational data on the utility of statin therapy in children with HeFH, showing a good safety profile, without liver toxicity side effects, no influence on growth trajectory and excellent efficacy in terms of LDL-C reduction over periods of 2-3 years [18][19][20][21].…”

“Apple does not fall far from the tree” – subclinical atherosclerosis in children with familial hypercholesterolemia.

“…Many researchers have already con rmed increased risk of atherosclerosis (also subclinical [20]) and subsequent cardiovascular events in adults with FH [21]. Studies concerning children with FH are not as numerous [15,22,23,24] but it is well documented that FH children have signi cantly increased IMCT in carotid arteries, femoral arteries and aorta when compared with healthy children as well as healthy siblings [24,25]. Although, there are discrepancies in age from which these differences become signi cant (between 10-12 for siblings [24,26] and 9-11 years for nonrelated controls [22,25]) and they can also be affected by treatment with statins.…”

Very special case of primary prevention with statins

“…A recent study has shown that children with familial hypercholesterolaemia (FH) commenced on statin therapy have a 1% incidence of cardiovascular events, compared with 27% in their affected parents who were treated with statins later in life after a 20 year follow‐up (Fig. ) …”

“…The investigation by Liurink et al . comes at apposite time when the value of statins in the primary prevention of coronary disease is again under scrutiny .…”