a Department of Pharmaceutical , Central Hospital of Linyi City , Yishui , Shandong , China.
Expert Rev Clin Pharmacol. 2019 Feb;12(2):95-99. doi: 10.1080/17512433.2019.1567326. Epub 2019 Jan 18.
Hereditary transthyretin-mediated amyloidosis is caused by a mutation in transthyretin (TTR) gene resulting in misfolded TTR protein accumulating as amyloid fibrils. Patisiran is a lipid nanoparticle formulation of ribonucleic acid interference (RNAi), which can reduce the production of TTR. Areas covered: In this review, the chemical property, mechanism of action, pharmacokinetics, clinical efficacy, and safety of patisiran were introduced. Expert Commentary: Patisiran offers a new treatment option for patients with hereditary transthyretin-mediated amyloidosis. Patisiran can significantly reduce the TTR level and improve patient's neuropathy and quality of life. The common adverse reactions were upper respiratory tract infections and infusion-related reactions.
遗传性转甲状腺素蛋白介导的淀粉样变性由转甲状腺素蛋白 (TTR) 基因突变引起，导致错误折叠的 TTR 蛋白积聚为淀粉样纤维。帕替沙林是一种小干扰 RNA（siRNA）的脂质纳米颗粒制剂，可降低 TTR 的产生。
在这篇综述中，介绍了帕替沙林的化学性质、作用机制、药代动力学、临床疗效和安全性。
帕替沙林为遗传性转甲状腺素蛋白介导的淀粉样变性患者提供了一种新的治疗选择。帕替沙林可显著降低 TTR 水平，改善患者的神经病变和生活质量。常见的不良反应是上呼吸道感染和输注相关反应。
