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。以新型CD20单克隆抗体、Bruton酪氨酸激酶(Bruton tyrosine kinase,BTK)抑制剂、磷脂腺肌醇3激酶(phosphoinositide 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单克隆抗体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)单克隆抗体、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。因此,新兴疗法为wAIHA患者的治疗提供了更多选择。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。
Abstract:
Warm autoimmune hemolytic anemia (wAIHA) is an autoimmune disorder mediated by autoantibodies. 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. Anti-B-cell targeted therapies, represented by novel CD20 monoclonal antibodies, Bruton tyrosine kinase (BTK) inhibitors, phosphoinositide 3-kinases (PI3K) inhibitors, and B-cell activating factor of the TNF family (BAFF) inhibitors, have shown remarkable 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. Consequently, these emerging therapies provide more options for the treatment of patients with wAIHA. This article reviews the recent advances in immunotargeted therapy for wAIHA, aiming to provide a reference for clinical practice.