Background The NICE 2008 Guidelines on the treatment and management of FH recommend that children with the disorder should be considered for statin treatment by the age of 10, and this advice has been reiterated in the 2013 NICE Quality Standards (QS41). There is still reluctance amongst some clinicians to start statins in children with FH at the age of 10 because of concerns about their long term safety. The Paediatric FH Register was established in 2012 in order to collect baseline and long term follow up data on all children with FH in UK.

Methods Paediatricians, and adult lipidologists seeing children, have been invited to enter baseline data on any child with a clinical diagnosis of FH using an electronic capture record. They are then prompted at 12 monthly intervals to update records with regard to lipid levels, growth trajectory and any side effects of statin use if prescribed.

Results So far we have baseline data on 71 children, 55% are boys and 75% are Caucasian. The mean age of the group is 12.7 years ranging from 3 to 18 years, with an untreated mean (+SD) total cholesterol of 7.91(+1.82) mmol/L and LDL of 5.95(+1.84) mmol/L. One individual had a total cholesterol >15 mmol/l compatible with a diagnosis of homozygous FH. 56% of the children were currently treated with statin and the treated group were older, (mean age 13.4 v 11.7 years) and had stronger evidence of a family history of early heart disease (38.5% have CHD in parent v 9.7% p = 0.007). The diagnostic LDL-cholesterol in those who were subsequently treated was marginally higher than in those not currently on statins 6.23(+2.14) mmol/L v 5.55(+1.25) mmol/L (p = 0.22). In statin-treated children mean LDL-cholesterol level was 1.97 mmol/L (32%) lower than in the untreated group: (4.2(+3.16) mmol/L compared to 5.75 (+2.67) mmol/L, p = 0.0002).

Conclusions The children registered so far have similar clinical characteristics to those identified in the 2012 UK FH National Audit. The data indicates that treatment decisions on children are being made based on having a stronger family history of CHD and higher LDL cholesterol.