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Review Article

治疗纯合子家族性高胆固醇血症的选择:洛米他派的作用

卷 27, 期 23, 2020

页: [3773 - 3783] 页: 11

弟呕挨: 10.2174/0929867326666190121120735

价格: $65

摘要

背景:洛米他派(美国Juxtapid®和欧洲Lojuxta®)是第一个开发的微粒体甘油三酯转移蛋白(MTP)的抑制剂，被批准为治疗纯合子家族性高胆固醇血症(HoFH)的新药物。它通过直接地或者选择性地与MTP结合，从而减少含脂蛋白的apo-B在肝脏和肠道中的合成与分泌。 目的:本文旨在总结洛米他派在治疗HoFH中的最新研究进展。 结果: 洛米他派的有效性和安全性已经在几个试验中进行了评估，并已显示其降低血浆中低密度脂蛋白胆固醇(LDL-C)水平平均超过50%。最常见的副作用是胃肠道和肝脏事件，但洛米他派通常表现出良好的耐受性和令人满意的患者依从性。最近，在欧洲，为了评估洛米他派长期的安全性和有效性，建立了the LOWER registry (ClinicalTrials.gov Identifier: NCT02135705)，以便从实际生活临床实践中获取用洛米他派 治疗患有HoFH患者的信息。此外，洛米他派降低甘油三酯水平的观察结果表明，可以考虑将洛米他派 用于治疗严重高甘油三酯血症患者——这些患者反复发作胰腺炎且对常规治疗反应不佳。 结论: 洛米他派是一种治疗HoFH的创新和有效的药物。长期安全数据、儿童和孕妇HoFH患者的治疗以及严重高甘油三酯血症的治疗仍需进一步研究。

关键词: 纯合子家族性高胆固醇血症，洛米他派，LOWER Registry，甘油三酯转移蛋白(MTP)抑制剂，MTP SNPs，常染色体显性高胆固醇血症(ADH)

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