骨髓增生异常综合征治疗药物——pevonedistat  

A therapeutic drug for myelodysplastic syndrome:pevonedistat

作　　者：王堂乐 王鹏 韩语轩 郭兆霖 曹爽[1] 刘彦 WANG Tang-le;WANG Peng;HAN Yu-xuan;GUO Zhao-lin;CAO Shuang;LIU Yan(School of Chemical Engineering and Pharmacy,Wuhan Institute of Technology,Wuhan 430205,China;Center for Drug Evaluation,National Medical Products Administration,Beijing 100022,China)

机构地区：[1]武汉工程大学化工与制药学院,武汉430205 [2]国家药品监督管理局药品审评中心,北京100022

出　　处：《临床药物治疗杂志》2024年第3期16-20,共5页Clinical Medication Journal

基　　金：武汉工程大学研究生教育创新基金(CX2022025)。

摘　　要：pevonedistat是一种新型神经前体细胞表达发育下调蛋白8(NEDD8)激活酶抑制剂,其可通过干扰NEDD8激活酶的生物活性破坏蛋白,从而导致肿瘤细胞死亡。pevonedistat对骨髓增生异常综合征及相关血液学肿瘤疾病的治疗效果已获得多个临床研究验证。本文对pevonedistat的基本信息、作用机制、药动学、临床疗效及安全性等进行综述,旨在为临床应用提供参考。Pevonedistat is a novel inhibitor of neuronal precursor cell-expressed developmentally down-regulated protein 8(NEDD8)activating enzyme.The drug disrupts the protein and causes tumor cell apoptosis by interfering with the biological activity of the NEDD8 activating enzyme.The therapeutic effect of pevonedistat on myelodysplastic syndrome and related hematological tumor diseases has been validated by multiple clinical studies.This article reviews the basic information,mechanism of action,pharmacokinetics,clinical efficacy,and safety of pevonedistat,aiming to provide a reference for clinical applications.

关 键 词：pevonedistat 骨髓增生异常综合征 急性髓系白血病 神经前体细胞表达发育下调蛋白8激活酶抑制剂 

分 类 号：R979.1[医药卫生—药品]