Alirocumab in Pediatric Patients With Heterozygous Familial Hypercholesterolemia
Santos R, Wiegman A, Caprio S, Cariou B, Averna M, Poulouin Y, Scemama M, Manvelian G, Garon G, Daniels S. Alirocumab in Pediatric Patients With Heterozygous Familial Hypercholesterolemia. JAMA Pediatrics 2024, 178: 283-293. PMID: 38315470, PMCID: PMC10845038, DOI: 10.1001/jamapediatrics.2023.6477.Peer-Reviewed Original ResearchDouble-blind periodLipid-lowering therapyHeterozygous familial hypercholesterolemiaPediatric patientsLDL-CLipid parametersFamilial hypercholesterolemiaRecommended low-density lipoprotein cholesterolAdjunctive lipid-lowering therapiesSecondary efficacy end pointsEnd pointsPediatric patients aged 8Least-squares mean differenceEfficacy of alirocumabOpen-label periodEfficacy end pointLow-density lipoprotein cholesterolReduced LDL-CBaseline to weekPrimary end pointAdverse event incidencePatients aged 8Randomized clinical trialsSquare mean differencesStatistically significant reduction