JAK1/2抑制剂治疗骨髓增殖性肿瘤的研究进展

Research progress of JAK1/2 inhibitors in the treatment of myeloproliferative neoplasms

bcr-abl阴性的骨髓增殖性肿瘤（MPN）包括真性红细胞增多症（PV）、原发性血小板增多症（ET）和原发性骨髓纤维化（PMF）.随着JAK2 V617F基因突变在MPN患者中的发现,一系列针对该突变的小分子靶向药物被研发,其中通过COMFORT-Ⅰ和COMFORT-Ⅱ等试验研究的JAK1/2抑制剂芦可替尼（ruxolitinib）已经被美国食品和药品管理局（FDA）和欧洲药品管理局（EMA）批准应用于中晚期PMF患者和羟基脲耐药或不耐受PV患者的治疗,给MPN患者带来了新的希望.

The bcr-abl negative myeloproliferative neoplasms （MPN） include polycythemia vera （PV）,essential thrombocythemia （ET） and primary myelofibrosis （PMF）.Along with the detecting of JAK2 V617F gene mutation in MPN patients,a series of small molecules drugs that targeted to the mutation have been investigated in clinical trials.Ruxolitinib is the inhibitor of JAK1/2,which has been proved by USA Food and Drug Administration （FDA） and the European Medicines Agency （EMA） to treat patients with intermediate-2 or high-risk PMF or post-PV MF or post-ET MF and PV patients who are intolerant or resistant to hydroxycarbamide,attributed to a number of clinical trials,especially in COMFORT-Ⅰ and COMFORT-Ⅱ.As a result,the MPN patients have new hope for the treatment.