Clinical management of children and young adults with heterozygous familial hypercholesterolaemia in the UK

Abstract

Life expectancy in familial hypercholesterolaemia (FH) has been greatly improved by the advent of statin therapy. In the UK, however, these agents are not licensed for use in children. We approached 169 physicians responsible for lipid clinics for information on their practice in young patients, and valid responses were received from 54%. A typical lipid clinic has only 3.5 patients aged under 16 with FH. In boys aged 10-15 years 65% of physicians were prepared to treat with bile acid sequestrants but only 23% with statins. There was greater reluctance to treat in girls of the same age, corresponding figures being 52% and 12%. Despite the efficacy of statins in reducing low-density-lipoprotein cholesterol, these agents are little used in children with FH. Their safety and clinical efficacy should be assessed by a randomized double-blind trial.

Figure 1

Cumulative frequency distribution of the age at which physicians would introduce lipid-lowering medication in young people with heterozygous familial hypercholesterolaemia

Figure 2

Cumulative frequency distribution of the level of serum cholesterol at which the physicians surveyed would introduce cholesterol-lowering medication in young people with heterozygous familial hypercholesterolaemia

Figure 3

Age distribution of patients in whom physicians would choose to use a bile acid sequestrating agent (BAS) on its own or statin treatment alone or in combination with a BAS agent. A few physicians use fenofibrate alone in younger children (not shown). ^*Alone or in combination