Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia: Phase 3, Multinational Open-Label Study

Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia: Phase 3, Multinational Open-Label Study

Despite progress in treating homozygous familial hypercholesterolemia, most patients do not achieve low-density lipoprotein cholesterol (LDL-C) targets. This study examined efficacy and safety of the PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitor, alirocumab, in pediatric patients (...

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Veröffentlicht in:Arteriosclerosis, thrombosis, and vascular biology thrombosis, and vascular biology, 2022-12, Vol.42 (12), p.1447-1457
Hauptverfasser: Bruckert, Eric, Caprio, Sonia, Wiegman, Albert, Charng, Min-Ji, Zárate-Morales, Cézar A., Baccara-Dinet, Marie T., Manvelian, Garen, Ourliac, Anne, Scemama, Michel, Daniels, Stephen R.
Format: Artikel
Sprache:eng
Schlagworte:
Adolescent
Antibodies, Monoclonal - adverse effects
Child
Cholesterol, LDL
Clinical and Population Studies
Double-Blind Method
Homozygous Familial Hypercholesterolemia
Humans
Life Sciences
Proprotein Convertase 9 - genetics
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Zusammenfassung:Despite progress in treating homozygous familial hypercholesterolemia, most patients do not achieve low-density lipoprotein cholesterol (LDL-C) targets. This study examined efficacy and safety of the PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitor, alirocumab, in pediatric patients (aged 8-17 years) with inadequately controlled homozygous familial hypercholesterolemia. In this open-label, single-arm, multinational, Phase 3 study, patients (n=18) received alirocumab 75 mg or 150 mg (bodyweight
ISSN:1079-5642
1524-4636
DOI:10.1161/ATVBAHA.122.317793