Quality statement 7: Drug treatment in children

Quality statement

Children with familial hypercholesterolaemia (FH) are assessed for lipid‑modifying drug treatment by a specialist with expertise in FH in a child‑focused setting by the age of 10 years.

Rationale

Children with FH begin to develop cardiovascular disease before clinical signs appear, with thickening of the carotid artery wall identifiable by the age of 10 years. Once a child is diagnosed as having FH, it is important they should be assessed for lipid‑modifying drug treatment as soon as possible. The assessment should include a discussion of the harms and benefits of different treatments. This allows children to start treatment as soon as it is appropriate and before significant atherosclerosis has developed.

Quality measures

Structure

Evidence of local arrangements to ensure that children with FH are assessed for lipid‑modifying drug treatment by a specialist with expertise in FH in a child‑focused setting by the age of 10 years.

Data source: Local data collection.

Process

Proportion of children with FH who are assessed for lipid‑modifying drug treatment by a specialist with expertise in FH in a child‑focused setting by the age of 10 years.

Numerator – The number of people in the denominator assessed for lipid‑modifying drug treatment by a specialist with expertise in FH in a child‑focused setting.

Denominator – The number of children with FH aged 10 years.

Data source: Local data collection.

What the quality statement means for service providers, healthcare practitioners, and commissioners

Service providers ensure that systems are in place for children with FH to be assessed for lipid‑modifying drug treatment by a specialist with expertise in FH in a child‑focused setting by the age of 10 years.

Specialists with expertise in FH in children and young people assess children with FH for lipid‑modifying drug treatment in a child‑focused setting by the age of 10 years.

Commissioners ensure that they commission services in which specialists with expertise in FH assess children with FH for lipid‑modifying drug treatment, in a child‑focused setting, by the age of 10 years.

What the quality statement means for patients, service users and carers

Children with FH have an assessment for possible drug treatment to reduce the low‑density cholesterol (bad cholesterol) in their blood by a specialist in a children's department, by the age of 10 years.

Source guidance

Familial hypercholesterolaemia: identification and management (NICE guideline CG71), recommendation 1.3.1.20

Definitions of terms used in this quality statement

Familial hypercholesterolaemia (FH)

FH relates to heterozygous FH only.

Specialist with expertise in FH in children and young people

A healthcare professional with expertise in FH in children and young people who has access to the wider skills of a multidisciplinary team. This team should include a dietitian, cardiologist and paediatrician, and a clinical genetic specialist to take a family history and obtain informed consent for a DNA test.

Assessment for lipid‑modifying drug treatment

NICE's guideline on familial hypercholesterolaemia recommends that the decision to offer or defer lipid‑modifying drug treatment for a child or young person should take into account their age, age at onset of coronary heart disease within the family and the presence of other cardiovascular risk factors, including their LDL‑C concentration.

Child‑focused setting

NICE's guideline on familial hypercholesterolaemia defines a child‑focused setting as valuing the child's view and validating their voice in making decisions impacting their lives. A child‑focused facility or space is one designed with the children's needs in mind.

Equality and diversity considerations

Boys and girls should have equal access to lipid‑modifying drug treatment for FH. There is anecdotal evidence that some clinicians are less likely to recommend statins in a girl than a boy at the same level of individual risk, arguing that because the age at onset of coronary heart disease in women is later than in men, the start of drug treatment can be 'safely' delayed until adulthood. However, many young women will need to stop statins for several years when they are trying to conceive, and during pregnancy and breastfeeding. Because the accumulation of LDL‑C burden is similar in boys and girls, this will result in the exposure to high LDL‑C being greater in early adulthood in young women than in their male siblings. Gender should not influence a clinician's decision to offer treatment; the decision should be made in accordance with the recommendations in NICE's guideline on familial hypercholesterolaemia, which indicate that lipid lowering with statins should be considered by the age of 10 years.