Nine-year overview of dyslipidemia management in children with heterozygous familial hypercholesterolemia: a university hospital outpatient lipid clinic project in Northwestern Greece.

Background To assess the efficacy and safety of lipid-lowering treatment in children with heterozygous familial hypercholesterolemia (HeFH) aged ≤12 years attending a tertiary hospital-based outpatient lipid clinic. Methods Data in 318 children from the University Hospital of Ioannina (Northwestern Greece) Outpatient Lipid Clinic Project for Children and Adolescents with Dyslipidemia from March 2009 to December 2018 were analyzed. We assessed the efficacy and safety treatment alongside any possible predictors of the achievement of the treatment target. Results Of 318 children with hyperlipidemia, 72 were diagnosed having HeFH based on clinical criteria and genetic confirmation. Compared with non-familial hypercholesterolemia (non-FH) children, those with FH had a higher occurrence of positive family history of premature cardiovascular disease, and higher levels of total, low-density lipoprotein-cholesterol (LDL-C), apolipoprotein B (apoB) and lipoprotein (a) (Lp(a)). Treatment regimens included either atorvastatin 10-20 mg/day, rosuvastatin 5-10 mg/day, pitavastatin 2-4 mg/day monotherapy or in combination with ezetimibe. The treatment goal of LDL-C (<135 mg/dL, 3.5 mmol/L) was achieved in 69% of children treated. The achievement of the treatment targets correlated positively with male sex and inversely with the Dutch Lipid Clinic Network Score, baseline total, LDL-C and apoB levels. No clinically significant changes in liver or muscle-related laboratory tests were reported; no effect on growth or sexual maturation was noted. Conclusions This study confirms that lipid-lowering treatment in HeFH children initiated in the setting of a specialized tertiary hospital-based outpatient lipid clinic is efficacious and safe. Children of male sex and low baseline lipid values had a better achievement of treatment target.