Nine-year overview of dyslipidemia management in children with heterozygous familial hypercholesterolemia: a university hospital outpatient lipid clinic project in Northwestern Greece
Abstract
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.
Author contributions: Thomas Benekos contributed to the analysis of data, participated in drafting the manuscript and approved the final version to be published and accepted responsibility for the entire content of the submitted manuscript. Chrysoula Kosmeri made substantial contributions to the analysis of data, participated in drafting the manuscript, approved the final version to be published and accepted responsibility for the entire content of the submitted manuscript. Antonios P Vlahos contributed to the conception of the study, critically revised and approved the final version of the manuscript to be published and accepted responsibility for the entire content of the submitted manuscript. Haralampos Milionis contributed to the design of the study and to the interpretation of data, supervised and edited the manuscript, approved the final version to be published and accepted responsibility for the entire content of the submitted manuscript.
Research funding: There is no funding resource.
Employment or leadership: None declared.
Honorarium: None declared.
Competing interests: The funding organization(s) played no role in the study design; in the collection, analysis, and interpretation of data; in the writing of the report; or in the decision to submit the report for publication.
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Articles in the same Issue
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- Letters to the Editor
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Articles in the same Issue
- Frontmatter
- Review
- Prevalence of obesity and overweight in Iranian students: a systematic review and meta-analysis
- Original Articles
- Trends in pediatric obesity management, a survey from the Pediatric Endocrine Society Obesity Committee
- Utility of MR proton density fat fraction and its correlation with ultrasonography and biochemical markers in nonalcoholic fatty liver disease in overweight adolescents
- Vitamin D status and its relation to insulin resistance in a Mexican pediatric population
- Elevated serum uric acid, hyperuricaemia and dietary patterns among adolescents in mainland China
- Is there a relationship between the dietary inflammatory index and metabolic syndrome among adolescents?
- New insights into the expression of androgen and estrogen receptors of the appendix testis in congenital cryptorchidism
- General properties of autoimmune thyroid diseases and associated morbidities
- Comparison of leptin levels in neonates born to mothers with high or low gestational weight gain
- Percentile values of serum zinc concentration and prevalence of its deficiency in Iranian children and adolescents: the CASPIAN-V study
- Nine-year overview of dyslipidemia management in children with heterozygous familial hypercholesterolemia: a university hospital outpatient lipid clinic project in Northwestern Greece
- Gesell Developmental Schedules scores and the relevant factors in children with Down syndrome
- Letters to the Editor
- Society for Pediatric Radiology (SPR) Child Abuse Committee response regarding Miller, Stolfi and Ayoub’s flawed theories about child abuse and metabolic bone disease
- Reply of Miller and Ayoub to Brown et al. Letter to the Editor
- Case Reports
- Identification of two novel variants in GAA underlying infantile-onset Pompe disease in two Pakistani families
- Hereditary vitamin D-resistant rickets: a report of four cases with two novel variants in the VDR gene and successful use of intermittent intravenous calcium via a peripheral route
- Variant analysis of HPD genes from two families showing elevated tyrosine upon newborn screening by tandem mass spectrometry (MS/MS)
- Challenges in the management of an ignored cause of hyperammonemic encephalopathy: pyruvate carboxylase deficiency
- Autoimmune polyglandular syndrome type II with co-manifestation of Addison’s and Graves’ disease in a 15-year-old boy: case report and literature review