Recommendation ID

What is the clinical effectiveness and safety of differing doses of lipid-modifying therapy in children with FH?

Any explanatory notes
(if applicable)

There have been no published studies to establish target serum LDL‑C concentration in treated children with FH receiving lipid-modifying drug therapy. Treatment is recommended from 10 years onwards, however this lack of data prevents a recommendation regarding the aim of pharmacological treatment on serum LDL‑C concentrations.

Research (both cross-sectional and longitudinal) should assess the evidence of end-organ involvement (for example, carotid intima medial thickness [IMT]) to determine at which age abnormalities can first be seen in children. The aim would be to identify a threshold effect, with an LDL‑C concentration below which carotid IMT is normal and where thickening is absent, and above which it is abnormal and where thickening is observed. Outcomes should include fasting serum total and LDL‑C concentration, carotid artery IMT, and growth and pubertal development.

Source guidance details

Comes from guidance
Familial hypercholesterolaemia: identification and management
Date issued
August 2008

Other details

Is this a recommendation for the use of a technology only in the context of research? No  
Is it a recommendation that suggests collection of data or the establishment of a register?   No  
Last Reviewed 31/10/2019