治疗复发性或难治性毛细胞白血病新药:帕克莫单抗

A new drug in treatment of relapsed or refractory hairy cell leukemia:moxetumomab pasudotox-tdfk

帕克莫单抗(moxetumomab pasudotox-tdfk)是由阿斯利康制药公司研发的一种以CD22为靶抗原的重组免疫毒素。2018年9月13日,美国食品和药物管理局批准帕克莫单抗用于治疗既往至少接受过2种系统治疗的复发性或难治性毛细胞白血病(HCL)患者。帕克莫单抗是首款针对性治疗HCL的抗体药物偶联物,其在清除微小残留病灶方面显著优于其他靶向治疗药物,如维罗非尼、依鲁替尼或利妥昔单抗。帕克莫单抗治疗相关的不良事件主要有恶心、外周性水肿、头痛和发热,患者一般均可良好耐受。

Moxetumomab pasudotox-tdfk(Mox-tox)is a recombinant immunotoxin with CD22 as target antigen developed by AstraZeneca Pharmaceuticals.It was approved for the treatment of patients with relapsed or refractory hairy cell leukemia(HCL)who had previously received at least two systemic therapies by the U.S.Food and Drug Administration on September 13,2018.Mox-tox is the first antibody drug conjugate for targeted treatment of HCL,which is significantly superior to other targeted drugs(such as vemurafenib,ibrutinib or rituximab)in clearing minimal residual disease.Mox-tox treatment-related adverse events mainly include nausea,peripheral edema,headache and fever,and patients are generally well tolerated.