JAK抑制剂在骨髓纤维化治疗中研究进展

Research Progress on Effect of JAK Inhibitors on Myelofibrosis——Review

骨髓纤维化(MF)是一种骨髓增殖性肿瘤,针对其治疗十分困难。随着JAK2V617F突变点的发现,JAK抑制剂为MF提供了新的治疗选择。自2011年美国FDA批准第一代JAK抑制剂Ruxolitinib上市以来,越来越多的JAK抑制剂进入临床试验,并取得了一定的临床效果。一方面,部分JAK抑制剂单药应用可有效减轻骨髓纤维化患者的临床症状,延缓病情进展,延长生存时间;另一方面,JAK抑制剂也可与传统药物或其他新型靶向药物联合应用,甚至作为异基因造血干细胞移植围移植期治疗用药,这些都为骨髓纤维化患者的治疗提供了更多的分子靶向治疗选择。本文主要介绍JAK抑制剂在骨髓纤维化治疗中的最新研究进展。

Myelofibrosis（ MF） is a type of myeloprolifirative neoplasms which is difficult to be treated. With the discovery of V617 F mutation site in Janus kinase 2（ JAK2）,JAK inhibitor provides a newtreatment strategy for patients with myelofibrosis. Since 2011 the FDA in USA approved the first generation of JAK inhibitor Ruxolitinib for marketing,a growing number of JAK inhibitors have been entering into the clinical trials and showed a certain clinical efficacy. On the one hand,some JAK inhibitors for single application can effectively relieve the clinical symptoms of patients with myelofibrosis,slowdown disease progression,and prolong the survival; on the other hand,JAK inhibitor can also be applied in combination with traditional or other newtargeted drugs for MF patients,even during the allogenetic hematopoietic stem cell transplantation,thus providing more choices for targeted therapy on the patients with myelofibrosis. This reviewfocuses mainly on the latest advances of JAK inhibitors for the patients with myelofibrosis.