氨己烯酸在婴儿痉挛症患儿中的有效性、安全性和个体化用药研究进展

    Research Progress on the Effectiveness, Safety, and Individualized Medication of Vigabatrin in Pediatric Patients with Infantile Spasms

    • 摘要: 氨己烯酸是2022年1月在中国上市的、针对婴儿痉挛症的一线治疗药物,它通过与γ-氨基丁酸转氨酶不可逆地结合,减少γ-氨基丁酸的降解而发挥抗癫痫发作作用。氨己烯酸具有特殊不良反应,特别是不可逆的视野缺损,为临床应用带来了挑战。笔者对国内外研究进行梳理,特别是对氨己烯酸的作用机制、给药方案、有效性、安全性、药动学以及影响暴露水平的因素等进行总结和讨论,以期为婴儿痉挛症患儿合理应用氨己烯酸提供参考。

       

      Abstract: Vigabatrin is a first-line treatment for infantile spasms that was launched in China in January 2022. It mainly exerts its antiepileptic seizure effects by irreversibly binding to γ-aminobutyric acid transaminase, thereby reducing the degradation of γ-aminobutyric acid. However, vigabatrin is associated with specific adverse reactions, particularly irreversible visual field defects, which pose challenges for its clinical application. Author provides a timely summary of research conducted both domestically and internationally, specifically discussing the mechanism of action, administration regimens, effectiveness, safety, pharmacokinetics, and factors affecting exposure levels of vigabatrin. The aim is to offer reference for the rational use of vigabatrin in pediatric patients with infantile spasms.

       

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