Treatment goal attainment in children with familial hypercholesterolemia: A cohort study of 302 children in Norway.

BACKGROUND

Consensus statements recommend that statin treatment in children with heterozygous familial hypercholesterolemia (FH) should be considered from 8 to 10 years of age. Although these recommendations are well known, less is known about actual treatment and treatment goal attainment in children with FH.

OBJECTIVE

The objective of the study was to investigate if children with FH were treated according to current recommendations.

METHODS

Retrospective collection of data from medical records of 302 children below 18 years visiting the Lipid Clinic, Oslo University hospital, during 2014 to 2016.

RESULTS

Ninety-nine percent had a genetically verified FH diagnosis. Mean age (standard deviation) at diagnosis, age at first visit, and time followed at the clinic was 8.5 (3.2), 9.5 (2.9), and 4.4 (2.7) years, respectively. Mean pretreatment low-density lipoprotein cholesterol (LDL-C) was 5.4 (1.4) mmol/L. Mean age at start of lipid-lowering therapy (LLT) was 12.5 (2.0) years, with no significant difference between girls and boys. LLT, mainly statins, was used by 177 (59%) children at their last visit. LDL-C in children treated with LLT was 3.6 (1.2) mmol/L (38% reduction from pretreatment, P < .001). A treatment goal of LDL-C ≤3.5 mmol/L was achieved by 43% of all children, by 58% of the children on LLT, and by 22% of children not on LLT.

CONCLUSION

Mean age at initiation of LLT is above the recommended 10 years of age and many children did not achieve the LDL-C treatment goal, even with follow-up at a dedicated lipid clinic. Earlier diagnosis and more frequent follow-ups are warranted.