Comparison of the characteristics at diagnosis and treatment of children with heterozygous familial hypercholesterolaemia (FH) from eight European countries

Autor: Kirsten B. Holven, Mafalda Bourbon, Tomáš Freiberger, Olivier S. Descamps, Susanne Greber-Platzer, Albert Wiegman, Jeanine E. Roeters van Lennep, Michal Vrablík, Gabriele Hanauer-Mader, Marta Futema, Euridiki Drogari, Uma Ramaswami, Hans Dieplinger, Martin Prøven Bogsrud, Steve E. Humphries
Přispěvatelé: Cardiology, Internal Medicine, Paediatric Metabolic Diseases, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism, ACS - Heart failure & arrhythmias, ACS - Diabetes & metabolism, ACS - Atherosclerosis & ischemic syndromes
Rok vydání: 2020
Předmět:
Male
0301 basic medicine
Heterozygote
Pediatrics
medicine.medical_specialty
Statin
Statin treatment
medicine.drug_class
Heterozygous familial hypercholesterolaemia
Paediatric FH
Age at diagnosis
Low density lipoprotein cholesterol
Heterozygous Familial Hypercholesterolaemia
030204 cardiovascular system & hematology
Article
Doenças Cardio e Cérebro-vasculares
Hyperlipoproteinemia Type II
LDL-C concentrations
03 medical and health sciences
0302 clinical medicine
Ezetimibe
Humans
Medicine
Child
Retrospective Studies
business.industry
Cholesterol
LDL
Statin Treatment
Treatment characteristics
3. Good health
Europe
030104 developmental biology
Lifestyle advice
Child
Preschool
Female
lipids (amino acids
peptides
and proteins)
Statin therapy
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Cardiology and Cardiovascular Medicine
business
LDL-C Concentrations
medicine.drug
Zdroj: Atherosclerosis, 292, 178-187. Elsevier Ireland Ltd
Atherosclerosis
ISSN: 0021-9150
DOI: 10.1016/j.atherosclerosis.2019.11.012
Popis: Background and aims For children with heterozygous familial hypercholesterolaemia (HeFH), European guidelines recommend consideration of statin therapy by age 8–10 years for those with a low density lipoprotein cholesterol (LDL-C) >3.5 mmol/l, and dietary and lifestyle advice. Here we compare the characteristics and lipid levels in HeFH children from Norway, UK, Netherlands, Belgium, Czech Republic, Austria, Portugal and Greece. Methods Fully-anonymized data were analysed at the London centre. Differences in registration and on treatment characteristics were compared by standard statistical tests. Results Data was obtained from 3064 children. The median age at diagnosis differed significantly between countries (range 3–11 years) reflecting differences in diagnostic strategies. Mean (SD) LDL-C at diagnosis was 5.70 (±1.4) mmol/l, with 88% having LDL-C>4.0 mmol/l. The proportion of children older than 10 years at follow-up who were receiving statins varied significantly (99% in Greece, 56% in UK), as did the proportion taking Ezetimibe (0% in UK, 78% in Greece). Overall, treatment reduced LDL-C by between 28 and 57%, however, in those >10 years, 23% of on-treatment children still had LDL-C>3.5 mmol/l and 66% of those not on a statin had LDL-C>3.5 mmol/l. Conclusions The age of HeFH diagnosis in children varies significantly across 8 countries, as does the proportion of those >10 years being treated with statin and/or ezetimibe. Approximately a quarter of the treated children and almost three quarters of the untreated children older than 10 years still have LDL-C concentrations over 3.5 mmol/l. These data suggest that many children with FH are not receiving the full potential benefit of early identification and appropriate lipid-lowering treatment according to recommendations.
Graphical abstract Image 1
Highlights • The age of HeFH diagnosis varies significantly between 8 European countries. • The proportion of HeFH children being treated varies across 8 European countries. • A quarter of FH children on statins have LDL-C above the target (>3.5 mmol/L). • Many FH children are not getting the full benefit of early diagnosis and treatment.
Databáze: OpenAIRE
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