Academic Journal
Management of Children with Heterozygous Familial Hypercholesterolemia Worldwide: A Meta-Analysis
| العنوان: | Management of Children with Heterozygous Familial Hypercholesterolemia Worldwide: A Meta-Analysis |
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| المؤلفون: | Bytyçi, Ibadete, Bytyqi, Sefer, Lewek, Joanna, Surma, Stanislaw, Bajraktari, Gani, Henein, Michael, Sahebkar, Amirhossein, Al-Khnifsawi, Mutaz, Gouni-Berthold, Ioanna, Pećin, Ivan, Toth, Peter P, Paneni, Francesco, Katsiki, Niki, Escobar, Carlos, Lavie, Carl J, Gaita, Dan, Santos, Raul D, Cicero, Arrigo F G, Bielecka-Dabrowa, Agata, Ahmed, Ali, Banach, Maciej |
| المصدر: | European Heart Journal Open ; ISSN 2752-4191 |
| بيانات النشر: | Oxford University Press (OUP) |
| سنة النشر: | 2025 |
| الوصف: | Background Heterozygous familial hypercholesterolemia (HeFH) is one of the most frequent monogenic disorders in the world, leading to premature atherosclerotic cardiovascular diseases (ASCVD). The aim of this meta-analysis was to evaluate the efficacy and safety of lipid lowering therapy (LLT) and achievement of low-density lipoprotein cholesterol (LDL-C) goal in children with HeFH. Methods The main endpoint was efficacy of goal achievement for LDL-C and other lipid parameters: total cholesterol [TC], triglycerides [TG], high density lipoprotein cholesterol [HDL-C], apolipoprotein B [apo B] and lipoprotein(a) [Lp(a)]), and the LLT safety (adverse events [AEs], including endocrine function, and growth indices). The secondary endpoint was an effect of LLT on attainment of LDL-C goal treatment (<3.5 mmol/L/130 mg/dL). Results A total of 41 studies with 4667 pediatric patients at mean age 12.08±2.4 years were included. 17 reported the efficacy and safety of LLT therapy compared to control, while the remaining assessed LLT through pre- and post-treatment. At median follow-up of 18.8 months, the group on LLT had significantly higher mean reductions of TC, LDL-C, TG, and increased HDL-C compared to control (-1.75 [-67,7 mg/dl], -1.84 [-71.2 mg/dl], -0.11 [-9.74 mg/dl], 0.08 mmol/L [3.1 mg/dl], respectively, p<0.001 for all). In the subgroup analysis according to different types of LLT we observed a significantly higher mean reduction of LDL-C by statin combined with ezetimibe treatment, followed by statins in monotherapy, PCSK9 inhibitors, and monotherapy with ezetimibe (-2.48 [-95.9 mg/dl], -2.16 [-83.5 mg/dl], -2.03 [-78.5 mg/dl], and -1.50 mmol/L [-58 mg/dl], respectively, test for overall effect: p<0.001). The pooled LDL-C was reduced by 33.44% (-2.14 mmol/L [-82.8 mg/dl], p<0.001) and failed to reach the goal treatment (<3.5 mmol/L) by 12.6% (95%CI, 12.4–12.9%). 38.7% of children achieved the LDL-C goal, 23.9% fell short by up to 10%, 10.7% experienced moderate failure ... |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| DOI: | 10.1093/ehjopen/oeaf001 |
| DOI: | 10.1093/ehjopen/oeaf001/61429665/oeaf001.pdf |
| الاتاحة: | https://doi.org/10.1093/ehjopen/oeaf001 https://academic.oup.com/ehjopen/advance-article-pdf/doi/10.1093/ehjopen/oeaf001/61429665/oeaf001.pdf |
| Rights: | https://creativecommons.org/licenses/by-nc/4.0/ |
| رقم الانضمام: | edsbas.1CF8CBBC |
| قاعدة البيانات: | BASE |
| DOI: | 10.1093/ehjopen/oeaf001 |
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