芦可替尼治疗费城染色体阴性骨髓增殖性肿瘤的临床研究进展

Clinical research progress of ruxolitinib in treatment of Philadelphia chromosomenegative myeloproliferative neoplasms

费城染色体阴性骨髓增殖性肿瘤(Ph-MPN)主要包括原发性骨髓纤维化(PMF)、真性红细胞增多症(PV)和原发性血小板增多症(PT)3种类型。自2011年,Janus激酶1/2(JAK1/2)抑制剂芦可替尼(ruxolitinib)已成为治疗PMF核心药物。此前异基因造血干细胞移植是唯一可能治愈PMF的治疗方法,但因没有配型或费用昂贵让多数患者却步。Ruxolitinib作为PMF的靶向药物,可以针对PMF的发病机制进行治疗,从根本上控制疾病,改善患者临床症状。Ruxolitinib除了可治疗PMF外,对羟基脲耐药或不耐药PV或PT患者亦起良好的作用。

Philadelphia chromosome-negative myeloproliferative neoplasms（ Ph-MPN） mainly include 3 types,that is,primary myelofibrosis（ PMF）,polycythemia vera（ PV）,and primary thrombocythemia（ PT）. The Janus kinase1/2（ JAK1/2） inhibitor ruxolitinib has been at the heart of PMF treatment since 2011. Before this,allogeneic hematopoietic stem cell transplantation is the only possible treatment for PMF,but it is prohibitive for most patients because of lack of matching or expensive aspects. As the targeted drug for PMF,ruxolitinib aims at the pathogenesis of PMF,so as to fundamentally control the disease and improve the clinical symptoms of patients. In addition to treating PMF,it also plays a good role in PV or PT patients with hydroxyurea resistance or intolerance.