2024
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
2023
Efficacy and safety of alirocumab in children and adolescents with heterozygous familial hypercholesterolaemia inadequately controlled with statins
Santos R, Wiegman A, Caprio S, Cariou B, Averna M, Poulouin Y, Scemama M, Manvelian G, Garon G, Daniels S. Efficacy and safety of alirocumab in children and adolescents with heterozygous familial hypercholesterolaemia inadequately controlled with statins. European Heart Journal 2023, 44: ehad655.2809. DOI: 10.1093/eurheartj/ehad655.2809.Peer-Reviewed Original ResearchProprotein convertase subtilisin/kexin type 9Safety of alirocumabKey secondary endpointHeterozygous familial hypercholesterolaemiaWeek 24Secondary endpointsPediatric patientsLipid parametersFamilial hypercholesterolaemiaTreatment periodTreatment groupsInternational phase 3 trialConvertase subtilisin/kexin type 9Subtilisin/kexin type 9Atherogenic lipid parametersEfficacy of alirocumabMean baseline LDLOpen-label periodPhase 3 trialLDL cholesterol levelsSignificant treatment differencesAlirocumab treatmentBaseline LDLPlacebo Q2WWeek 104