دورية أكاديمية
Alirocumab in Pediatric Patients With Heterozygous Familial Hypercholesterolemia ; A Randomized Clinical Trial
| العنوان: | Alirocumab in Pediatric Patients With Heterozygous Familial Hypercholesterolemia ; A Randomized Clinical Trial |
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| المؤلفون: | Santos, Raul D., Wiegman, Albert, Caprio, Sonia, Cariou, Bertrand, Averna, Maurizio, Poulouin, Yann, Scemama, Michel, Manvelian, Garen, Garon, Genevieve, Daniels, Stephen |
| المصدر: | JAMA Pediatrics ; volume 178, issue 3, page 283 ; ISSN 2168-6203 |
| بيانات النشر: | American Medical Association (AMA) |
| سنة النشر: | 2024 |
| الوصف: | Importance Many pediatric patients with heterozygous familial hypercholesterolemia (HeFH) cannot reach recommended low-density lipoprotein cholesterol (LDL-C) concentrations on statins alone and require adjunct lipid-lowering therapy (LLT); the use of alirocumab in pediatric patients requires evaluation. Objective To assess the efficacy of alirocumab in pediatric patients with inadequately controlled HeFH. Design, Setting, and Participants This was a phase 3, randomized clinical trial conducted between May 2018 and August 2022 at 43 centers in 24 countries. Pediatric patients aged 8 to 17 years with HeFH, LDL-C 130 mg/dL or greater, and receiving statins or other LLTs were included. Following consecutive enrollment into dosing cohorts, 25 of 99 patients screened for dosing every 2 weeks (Q2W) failed screening; 25 of 104 patients screened for dosing every 4 weeks (Q4W) failed screening. A total of 70 of 74 Q2W patients (95%) and 75 of 79 Q4W patients (95%) completed the double-blind period. Interventions Patients were randomized 2:1 to subcutaneous alirocumab or placebo and Q2W or Q4W. Dosage was based on weight (40 mg for Q2W or 150 mg for Q4W if <50 kg; 75 mg for Q2W or 300 mg for Q4W if ≥50 kg) and adjusted at week 12 if LDL-C was 110 mg/dL or greater at week 8. After the 24-week double-blind period, patients could receive alirocumab in an 80-week open-label period. Main Outcomes and Measures The primary end point was percent change in LDL-C from baseline to week 24 in each cohort. Results Among 153 patients randomized to receive alirocumab or placebo (mean [range] age, 12.9 [8-17] years; 87 [56.9%] female), alirocumab showed statistically significant reductions in LDL-C vs placebo in both cohorts at week 24. Least squares mean difference in percentage change from baseline was −43.3% (97.5% CI, −56.0 to −30.7; P < .001) Q2W and −33.8% (97.5% CI, −46.4 to −21.2; P < .001) Q4W. Hierarchical analysis of secondary efficacy end points demonstrated significant improvements in other ... |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| DOI: | 10.1001/jamapediatrics.2023.6477 |
| الإتاحة: | https://doi.org/10.1001/jamapediatrics.2023.6477Test https://jamanetwork.com/journals/jamapediatrics/articlepdf/2814614/jamapediatrics_santos_2024_oi_230095_1708531145.6393.pdfTest |
| رقم الانضمام: | edsbas.F2234937 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1001/jamapediatrics.2023.6477 |
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