温抗体型自身免疫性溶血性贫血的免疫靶向治疗进展  

Advances in Immunotargeted Therapy for Warm Autoimmune Hemolytic Anemia

作　　者：王琦[1] 陈苗[1] WANG Qi;CHEN Miao(Department of Hematology,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100730,China)

机构地区：[1]中国医学科学院北京协和医学院北京协和医院血液内科,北京100730

出　　处：《协和医学杂志》2025年第2期423-430,共8页Medical Journal of Peking Union Medical College Hospital

基　　金：中央高水平医院临床科研专项(2022-PUMCH-B-048)。

摘　　要：温抗体型自身免疫性溶血性贫血(warm autoimmune hemolytic anemia,wAIHA)是由自身抗体介导的自身免疫性疾病。随着对wAIHA免疫发病机制的深入理解,针对免疫系统不同靶点的药物研发取得了显著进展,为wAIHA患者的治疗提供了更多选择。以新型CD20单抗、Bruton酪氨酸激酶(Bruton tyrosine kinase,BTK)抑制剂、磷脂酰肌醇3激酶(phosphatidylinositol 3-kinases,PI3K)抑制剂和B淋巴细胞活化因子(B-cell activating factor of the TNF family,BAFF)抑制剂等为代表的抗B细胞靶向治疗,以及以蛋白酶体抑制剂和CD38单抗为代表的抗浆细胞靶向治疗均取得了显著成效。此外,补体抑制剂、新生儿Fc受体(neonatal Fc receptor,FcRn)单抗、脾酪氨酸激酶(spleen tyrosine kinase,SYK)抑制剂、哺乳动物雷帕霉素靶蛋白(mammalian target of rapamycin,mTOR)抑制剂等也取得了显著进展。本文对近年来wAIHA的免疫靶向治疗进展进行综述,以期为临床实践提供参考。Warm autoimmune hemolytic anemia(wAIHA)is an autoimmune disorder mediated by au⁃toantibodies.With an increasing understanding of the immunopathogenesis of wAIHA,significant progress has been made in the development of drugs targeting different components of the immune system.Consequently,these emerging drugs provide more options for the treatment of patients with wAIHA.Anti⁃B⁃cell targeted therapies,re⁃presented by novel CD20 monoclonal antibodies,Bruton tyrosine kinase(BTK)inhibitors,phosphatidylinositol 3⁃kinases(PI3K)inhibitors,and B⁃cell activating factor of the TNF family(BAFF)inhibitors,have shown re⁃markable efficacy.Additionally,anti⁃plasma cell targeted therapies,exemplified by proteasome inhibitors and CD38 monoclonal antibodies,have also demonstrated significant outcomes.Furthermore,advances have been achieved in complement inhibitors,neonatal Fc receptor(FcRn)monoclonal antibodies,spleen tyrosine kinase(SYK)inhibitors,and mammalian target of rapamycin(mTOR)inhibitors.This article reviews the recent advances in immunotargeted therapy for wAIHA,aiming to provide a reference for clinical practice.

关 键 词：温抗体型 自身免疫性溶血性贫血 免疫治疗 靶向治疗 

分 类 号：R556.6[医药卫生—血液循环系统疾病] R979.1[医药卫生—内科学]