PCSK9 inhibition with alirocumab in pediatric patients with heterozygous familial hypercholesterolemia: The ODYSSEY KIDS study

Heterozygous familial hypercholesterolemia (HeFH) is a genetic disorder characterized by elevated levels of low-density lipoprotein cholesterol (LDL-C). This phase 2 dose-finding study (NCT02890992) evaluated the efficacy, safety, and dose selection of proprotein convertase subtilisin/kexin type 9 (...

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Bibliographic Details
Published in:Journal of clinical lipidology 2020-05, Vol.14 (3), p.322-330.e5
Main Authors: Daniels, Stephen, Caprio, Sonia, Chaudhari, Umesh, Manvelian, Garen, Baccara-Dinet, Marie T., Brunet, Aurelie, Scemama, Michel, Loizeau, Virginie, Bruckert, Eric
Format: Article
Language:English
Subjects:
Alirocumab
Cardiovascular
Heterozygous familial hypercholesterolemia
Life Sciences
PCSK9 inhibitor
Pediatrics
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Summary:Heterozygous familial hypercholesterolemia (HeFH) is a genetic disorder characterized by elevated levels of low-density lipoprotein cholesterol (LDL-C). This phase 2 dose-finding study (NCT02890992) evaluated the efficacy, safety, and dose selection of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor alirocumab in pediatric HeFH patients. HeFH patients (n = 42) who were aged 8–17 years, had body weight (BW) ≥25 kg, and had LDL-C ≥130 mg/dL despite optimal statin/other lipid-modifying therapies were enrolled in 4 cohorts according to BW: cohort #1: 30 mg (
ISSN:1933-2874
1876-4789
DOI:10.1016/j.jacl.2020.03.001