Mohamed Nasser, Hazem E Mohammed, Mohamed E Haseeb, Mohamed Khalafalla Darwish, George Hanen, Nada A Abdelaziz, Anas Hussien Heiba, Abdelrahman Shata, Mohamed Salem Abdelkader
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We aimed to evaluate the efficacy and safety of lomitapide in patients with HoFH through a systematic review and meta-analysis of available clinical evidence.</p><p><strong>Methods: </strong>A comprehensive search was conducted in PubMed, Scopus, and Web of Science through March 2025. Observational studies and clinical trials reporting on lipid profile changes and safety outcomes in HoFH patients receiving lomitapide were included. Outcomes were pooled using random-effects models, and heterogeneity was assessed using the I<sup>2</sup> statistic.</p><p><strong>Results: </strong>Eight studies comprising both adult and pediatric patients (n = 209) were included. Lomitapide significantly reduced LDL-C levels by 49.27%, total cholesterol by 46.05%, and apolipoprotein B by 51.01%. Reductions were also observed in triglycerides, VLDL-C, and non-HDL-C. HDL-C remained relatively unchanged. Adverse events were mostly gastrointestinal, with a 14% discontinuation rate. The overall quality of studies ranged from fair to good.</p><p><strong>Conclusions: </strong>Lomitapide demonstrates substantial efficacy in reducing LDL-C and other atherogenic lipids in HoFH patients, with an acceptable safety profile. These findings support its role as an adjunctive therapy in this population, though further randomized controlled trials are warranted to validate long-term safety and effectiveness.</p>","PeriodicalId":9557,"journal":{"name":"Cardiovascular Drugs and Therapy","volume":" ","pages":""},"PeriodicalIF":3.1000,"publicationDate":"2025-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Lomitapide, a Microsomal Triglyceride Transfer Protein Inhibitor, in Homozygous Familial Hypercholesterolemia: A Systematic Review and Meta-Analysis of Efficacy and Safety.\",\"authors\":\"Mohamed Nasser, Hazem E Mohammed, Mohamed E Haseeb, Mohamed Khalafalla Darwish, George Hanen, Nada A Abdelaziz, Anas Hussien Heiba, Abdelrahman Shata, Mohamed Salem Abdelkader\",\"doi\":\"10.1007/s10557-025-07764-4\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Purpose: </strong>Homozygous Familial Hypercholesterolemia (HoFH) is a rare and life-threatening genetic disorder characterized by elevated low-density lipoprotein cholesterol (LDL-C) and early-onset atherosclerotic cardiovascular disease. 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引用次数: 0
摘要
目的：纯合子家族性高胆固醇血症（HoFH）是一种罕见且危及生命的遗传性疾病，其特征是低密度脂蛋白胆固醇（LDL-C）升高和早发性动脉粥样硬化性心血管疾病。Lomitapide是一种微粒体甘油三酯转移蛋白（MTP）抑制剂，可以独立于LDL受体功能降低LDL- c，为该人群提供了一种替代治疗方法。我们旨在通过对现有临床证据的系统回顾和荟萃分析来评估洛米他胺对HoFH患者的疗效和安全性。方法：综合检索PubMed、Scopus和Web of Science，截止到2025年3月。观察性研究和临床试验报告了接受洛米他胺治疗的HoFH患者的血脂变化和安全性结果。使用随机效应模型汇总结果，并使用I2统计量评估异质性。结果：包括成人和儿童患者的8项研究（n = 209）被纳入。Lomitapide显著降低LDL-C水平49.27%，总胆固醇降低46.05%，载脂蛋白B降低51.01%。甘油三酯、VLDL-C和非hdl - c也有降低。HDL-C保持相对不变。不良事件主要发生在胃肠道，停药率为14%。研究的总体质量从一般到良好不等。结论：在降低HoFH患者的LDL-C和其他致动脉粥样硬化性脂质方面，洛米他啶具有显著的疗效，并具有可接受的安全性。尽管需要进一步的随机对照试验来验证其长期安全性和有效性，但这些发现支持其作为辅助治疗在该人群中的作用。
Lomitapide, a Microsomal Triglyceride Transfer Protein Inhibitor, in Homozygous Familial Hypercholesterolemia: A Systematic Review and Meta-Analysis of Efficacy and Safety.
Purpose: Homozygous Familial Hypercholesterolemia (HoFH) is a rare and life-threatening genetic disorder characterized by elevated low-density lipoprotein cholesterol (LDL-C) and early-onset atherosclerotic cardiovascular disease. Lomitapide, a microsomal triglyceride transfer protein (MTP) inhibitor, decreases LDL-C independent of LDL receptor function, providing an alternative treatment in this population. We aimed to evaluate the efficacy and safety of lomitapide in patients with HoFH through a systematic review and meta-analysis of available clinical evidence.
Methods: A comprehensive search was conducted in PubMed, Scopus, and Web of Science through March 2025. Observational studies and clinical trials reporting on lipid profile changes and safety outcomes in HoFH patients receiving lomitapide were included. Outcomes were pooled using random-effects models, and heterogeneity was assessed using the I2 statistic.
Results: Eight studies comprising both adult and pediatric patients (n = 209) were included. Lomitapide significantly reduced LDL-C levels by 49.27%, total cholesterol by 46.05%, and apolipoprotein B by 51.01%. Reductions were also observed in triglycerides, VLDL-C, and non-HDL-C. HDL-C remained relatively unchanged. Adverse events were mostly gastrointestinal, with a 14% discontinuation rate. The overall quality of studies ranged from fair to good.
Conclusions: Lomitapide demonstrates substantial efficacy in reducing LDL-C and other atherogenic lipids in HoFH patients, with an acceptable safety profile. These findings support its role as an adjunctive therapy in this population, though further randomized controlled trials are warranted to validate long-term safety and effectiveness.
期刊介绍：
Designed to objectively cover the process of bench to bedside development of cardiovascular drug, device and cell therapy, and to bring you the information you need most in a timely and useful format, Cardiovascular Drugs and Therapy takes a fresh and energetic look at advances in this dynamic field.
Homing in on the most exciting work being done on new therapeutic agents, Cardiovascular Drugs and Therapy focusses on developments in atherosclerosis, hyperlipidemia, diabetes, ischemic syndromes and arrhythmias. The Journal is an authoritative source of current and relevant information that is indispensable for basic and clinical investigators aiming for novel, breakthrough research as well as for cardiologists seeking to best serve their patients.
Providing you with a single, concise reference tool acknowledged to be among the finest in the world, Cardiovascular Drugs and Therapy is listed in Web of Science and PubMed/Medline among other abstracting and indexing services. The regular articles and frequent special topical issues equip you with an up-to-date source defined by the need for accurate information on an ever-evolving field. Cardiovascular Drugs and Therapy is a careful and accurate guide through the maze of new products and therapies which furnishes you with the details on cardiovascular pharmacology that you will refer to time and time again.
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