复发/难治性温抗体型自身免疫性溶血性贫血患者治疗的研究进展  

Research progress on treatment of patients with relapsed/refractory warm antibody autoimmune hemolytic anemia

作　　者：史忆萌 张凤奎 Shi Yimeng;Zhang Fengkui(State Key Laboratory of Experimental Hematology,National Clinical Research Center for Blood Diseases,Haihe Laboratory of Cell Ecosystem,Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Tianjin 300020,China)

机构地区：[1]中国医学科学院血液病医院(中国医学科学院血液学研究所),实验血液学国家重点实验室,国家血液系统疾病临床医学研究中心,细胞生态海河实验室,天津300020

出　　处：《国际输血及血液学杂志》2024年第1期69-74,共6页International Journal of Blood Transfusion and Hematology

摘　　要：温抗体型自身免疫性溶血性贫血(wAIHA)是由于最适结合温度为37℃的自身抗体与红细胞膜上的抗原结合,使红细胞被过早破坏所致的贫血。wAIHA患者一线糖皮质激素治疗的总体反应率(ORR)为80%~90%,但仅有20%~30%患者在激素减量或停药后获得持续缓解。对激素治疗无效或复发wAIHA患者的推荐药物为利妥昔单抗,但仍有>25%患者面临再次复发或二线治疗失败。传统的非靶向免疫抑制剂及脾切除术的疗效不确定且不良反应严重,仅作为三线或后线治疗方案。因此,目前复发/难治性wAIHA(R/R wAIHA)患者的治疗选择非常有限,整体疗效及预后不佳。笔者拟就针对B、T及浆细胞的治疗,抑制Fcγ受体(FcγR)信号转导的治疗,以及新生儿Fc受体(FcRn)抑制剂等,在R/R wAIHA患者治疗中的研究进展进行综述,旨在为R/R wAIHA患者的药物治疗策略和进一步的临床研究提供参考。Warm active antibody autoimmune hemolytic anemia(wAIHA)is a kind of anemia caused by premature destruction of erythrocytes due to binding of autoantibodies to antigens on the erythrocyte membrane at an optimal binding temperature of 37°C.The overall response rate(ORR)of first-line glucocorticoid therapy in patients with wAIHA is 80%-90%,but only 20%-30%of patients achieve sustained remission after glucocorticoid taper or discontinuation.Rituximab therapy is recommended for glucocorticoid-naïve or relapsed patients,but>25%of patients still face relapse or failure of second-line therapy.Conventional non-targeted immunosuppressive agents and splenectomy have uncertain efficacy and serious adverse reactions,and are only used as third-or back-line treatment options.Therefore,the current treatment options for patients with relapsed/refractory wAIHA(R/R wAIHA)are very limited,and the overall outcome and prognosis are poor.This article reviews the research progress in the treatment of patients with R/R wAIHA,including treatments targeting B,T and plasma cells,treatments that inhibit Fcγreceptor(FcγR)signal transduction,and neonatal Fc receptor(FcRn)inhibitors,etc.,aiming to provide a reference for drug treatment strategies and further clinical research in patients with R/R wAIHA.

关 键 词：贫血 溶血性 自身免疫性 脾酪氨酸激酶抑制剂 Bruton酪氨酸激酶抑制剂 新生儿Fc受体抑制剂 长寿命浆细胞 

分 类 号：R556.62[医药卫生—血液循环系统疾病]