用于治疗骨髓纤维化的Janus活化激酶2/Fms样酪氨酸激酶3抑制剂新药帕克替尼  

Pacritinib:a new Janus activated kinase 2/Fms-like tyrosine kinase 3 inhibitor for the treatment of myelofibrosis

作　　者：王咏懿 黄璐 纪雪梅[4] WANG Yong-yi;HUANG Lu;JI Xue-mei(School of Pharmacy,China Pharmaceutical University,Nanjing 211198,China;School of Chemical Engineering and Pharmacy,Wuhan Institute of Technology,Wuhan 430205,China;School of Pharmaceutical Sciences,Wuhan University,Wuhan 430071,China;College of Life Sciences and Technology,China Pharmaceutical University,Nanjing 211198,China)

机构地区：[1]中国药科大学药学院,南京211198 [2]武汉工程大学化工与制药学院,武汉430205 [3]武汉大学药学院,武汉430071 [4]中国药科大学生命科学与技术学院,南京211198

出　　处：《中国新药杂志》2023年第10期994-999,共6页Chinese Journal of New Drugs

摘　　要：骨髓纤维化(MF)是一种骨髓增殖性肿瘤,常伴有血小板减少症,严重影响患者的生活质量并存在向白血病转化的风险。帕克替尼是一种Janus活化激酶2(JAK2)及Fms样酪氨酸激酶3(FLT3)抑制剂。帕克替尼胶囊(商品名:Vonjo)于2022年2月28日被美国FDA加速批准,用于治疗患有罕见形式的骨髓疾病(中危或高危的原发性或继发性MF)且血小板(凝血细胞)水平低于50000·μL^(-1)的成人患者,口服200 mg,bid的剂量显示出良好的临床疗效和可控的安全性。本文对其作用机制、药动学、药效学、临床疗效及安全性进行综述,为临床应用提供参考。Myelofibrosis(MF)is a myeloproliferative neoplasms(MPN),usually accomplies with thrombocytopenia,severely impacts the life quality of patients and presents a risk for leukemic transformation.Pacritinib is a kind of Janus activated kinase 2(JAK2)and Fms-like tyrosine kinase 3(FLT3)inhibitor.U.S.FDA has granted accelerated approval for pacritinib capsules(Vonjo),on Feb 28,2022,in the treatment of adult patients with the platelet(blood clotting cells)levels below 50000·μL^(-1)who have a rare form of bone marrow disorder known as intermediate or high-risk primary and secondary myelofibrosis.The oral dose of 200 mg twice daily showed good clinical efficacy and manageable safety.In this paper,the mechanism of action,pharmacodynamics,pharmacokinetics,safety,and clinical efficacy of daridorexant are reviewed.

关 键 词：骨髓纤维化 Janus活化激酶2/Fms样酪氨酸激酶3抑制剂 帕克替尼 骨髓增殖性肿瘤 血小板减少症 临床疗效 安全性 

分 类 号：R979.1[医药卫生—药品]