早老样核纤层蛋白病治疗药物:lonafarnib  

A drug in treatment of progeroid laminopathies:lonafarnib

作　　者：张文竹[1] 苗莹[1] 孙韬华[1] 张媛媛[1] ZHANG Wen-zhu;MIAO Ying;SUN Tao-hua;ZHANG Yuan-yuan(Department of Drug Clinical Trials,Qingdao Municipal Hospital,Qingdao SHANDONG 266011,China)

机构地区：[1]青岛市市立医院药物临床试验研究科,山东青岛266011

出　　处：《中国新药与临床杂志》2022年第1期17-20,共4页Chinese Journal of New Drugs and Clinical Remedies

摘　　要：lonafarnib是一种法尼基转移酶抑制剂,可抑制早老蛋白的法尼基化以及早老蛋白和早老蛋白样蛋白在核内膜的积累。2020年11月,lonafarnib获美国食品和药物管理局批准,用于降低Hutchinson-Gilford综合征的死亡风险及治疗缺陷型早老样核纤层蛋白病;接受治疗的患者应年龄≥12个月,体表面积≥0.39 m2。临床试验表明lonafarnib可改善患者体重,增加骨密度,延长平均寿命,常见不良反应包括呕吐、腹泻、感染等。Lonafarnib is a farnesyltransferase inhibitor that can inhibit the farnesylation and subsequent accumulation of progerin and progerin-like proteins in the inner nuclear membrane.In November 2020,lonafarnib was approved by the U.S.Food and Drug Administration to reduce the risk of mortality in Hutchinson-Gilford progeria syndrome,as well as for treatment of processing-defcient progeroid laminopathies.The patients received lonafarnib should be aged≥12 months and have a body surface area≥0.39 m2.Clinical trials have shown that lonafarnib can improve patient weight,increase bone density,and extend average life span.The common adverse reactions include vomiting,diarrhea,infection and so on.

关 键 词：LONAFARNIB 早衰 法尼基转移酶 早老样核纤层蛋白病 

分 类 号：R592[医药卫生—老年医学]