鲁索替尼的临床应用研究进展  

Research progress on clinical application of ruxolitinib

作　　者：冯世权 秦贞苗[1] 胡雪 董德峤 彭浩洋 甘昌冉 段成成 高亚男 FENG Shiquan;QIN Zhenmiao;HU Xue;DONG Deqiao;PENG Haoyang;GAN Changran;DUAN Chengcheng;GAO Yanan(College of Pharmacy,Hainan Medical University,Haikou 571199,China)

机构地区：[1]海南医学院药学院,海口571199

出　　处：《中国药房》2024年第13期1668-1672,共5页China Pharmacy

基　　金：国家自然科学基金项目(No.82060642);海南省自然科学基金高层次人才项目(No.821RC569);海南省高等学校科学研究项目(No.Hnky2020-35);海南省科协青年科技英才创新计划项目(No.QCXM202029)。

摘　　要：鲁索替尼是一种选择性靶向Janus激酶(JAK)的小分子抑制剂,可在JAK1、JAK2结构域与催化位点上的三磷酸腺苷竞争性结合,从而抑制JAK活化和信号转导及转录激活蛋白(STAT)磷酸化,阻止JAK-STAT信号通路的表达。口服鲁索替尼对骨髓纤维化和真性红细胞增多症展现出良好的疗效。外用的鲁索替尼乳膏是美国FDA批准的首个非节段型白癜风家庭治疗药物,目前虽尚未在我国上市,但已于2023年8月落地国内医疗先行区,有望成为白癜风治疗的新突破。鲁索替尼乳膏还被多项临床案例证明对特应性皮炎、斑秃等具有显著疗效,具有较大的应用前景。Ruxolitinib,a small molecule inhibitor,selectively targets Janus kinase(JAK)by competitively binding to adenosine triphosphate on the catalytic site of the JAK1 and JAK2 domain,thereby inhibiting JAK activation and signal transducer and activator of transcription(STAT)phosphorylation and prevents the expressions of the JAK-STAT signaling pathway.Oral ruxolitinib has demonstrated promising efficacy for myelofibrosis and polycythemia vera.The topical Ruxolitinib cream,approved by the US FDA as the first non-segmental vitiligo home treatment drug,is set to be launched in domestic medical pioneer areas in August 2023 and is expected to bring about a breakthrough in the treatment of vitiligo.Clinical cases have also shown that Ruxolitinib cream has significant curative effects on atopic dermatitis,alopecia areata,and other conditions,indicating great application prospects.

关 键 词：鲁索替尼 JAK-STAT信号通路 JAK抑制剂 骨髓纤维化 真性红细胞增多症 白癜风 皮肤病 

分 类 号：R961[医药卫生—药理学] R969[医药卫生—药学]