Statin use effective and safe in children with familial hypercholesterolaemia
A recent study using data from the UK Paediatric Familial Hypercholesterolaemia Register shows that statin therapy reduces LDL cholesterol by 31% with no associated liver or muscle damage. Statin use in children is not associated with any reductions in growth rate and is safe in childhood, with no biochemical evidence of toxicity over a 2- to 3-year period.
The study set out to assess the prevalence of plasma markers of liver toxicity and muscle damage in statin-treated FH children; the prevalence of obesity in FH children compared to the UK general population, and to compare growth rates in statin-treated and non-treated children.
The most commonly used statins were atorvastatin (49.2%; n = 63), pravastatin (27.3%, n = 35), simvastatin (21.1%; n = 27), and rosuvastatin (2.3%; n = 3). In the register, there are 71 children > 10 years of age had not started on statin therapy. Of these, over 80% had LDL-C >3.5 mmol/L, and 35% had evidence of early CHD in a first-degree relative. As such, these children would be strong candidates for statin treatment, following recent European expert opinion guidelines (Weigman A et al., Eur Heart J 2015;36:2425).
In the statin-treated children, although LDL-C levels had been lowered by 31%, over half (55.6%) still had levels over the suggested target of 3.5 mmol/L. The authors suggested this could be due to poor adherence and a clinical decision to postpone the uptitration of statin dose for safety reasons until a child is older.
All FH guidelines recommend a healthy low-fat diet and exercise lifestyle as part of the management programme for children (and adults) with FH, and it appears that this advice is being followed in the children seen in the Registry. In addition, none of children had type 1 or type 2 diabetes which is a known risk in non-FH children treated with statins.
- Key study results
- 300 children with FH (51% boys, 75% Caucasian, untreated mean [SD] LDL-C 5.50 [1.49] mmol/L)
- Proportion on statins varied significantly by age group (15 years = 73.2%)
- Statin treatment reduced LDL-C by 31% (1.84 [1.43] mmol/L), with no elevated liver toxicity markers or muscle damage.
- At registration, 16.9% of FH children were overweight (similar to non-FH children) and 11.1% were obese (vs 21.2% in non-FH children).
- No difference in annual growth rate in statin vs no-statin groups (age-adjusted weight increases 3.58 vs 3.53 kg; P=0.91, height 4.45 vs 4.60 cm, P=0.73).
The majority of the children in the Register were identified by family studies from an index case with a clinical diagnosis of FH. These results show the importance of early referral to specialist treatment for these children and the safety and effectiveness of statins in these patients.
Humphries SE, Cooper J, Ramaswami U, et al. The UK paediatric familial hypercholesterolaemia register: statin-related safety and 1-year growth data. J Clin Lipidol 2017;pii: S1933-2874(17)30514-7.