儿童异基因造血干细胞移植后自身免疫性溶血性贫血的研究进展

Progress of autoimmune hemolytic anemia after allogeneic hematopoietic stem cell transplantation in children

在进行异基因造血干细胞移植(allogeneic hemopoietic stem cell transplantation,allo-HSCT)的儿童患者中,自身免疫性溶血性贫血(autoimmune hemolytic anemia,AIHA)的发病率为2%~6%,危险因素包括移植时年龄较小、非肿瘤性疾病、无关供者移植、淋巴细胞耗竭药物的使用和慢性移植物抗宿主病等,这些危险因素的共同特点是移植后不完全的免疫重建或免疫失调,可能与发病机制相关。移植后AIHA的治疗具有挑战性,目前尚无标准化的治疗指南,一线治疗仍为激素,但激素治疗的复发率高,完全缓解率约为30%,其他常规治疗如静脉用免疫球蛋白、血浆置换和脾切除通常对移植后AIHA无效。近年来,一些研究尝试使用单克隆抗体、免疫抑制剂等药物作为二、三线治疗方案,取得了较高的缓解率,但由于研究的病例数有限,其持续缓解率尚不能确定。该文对移植后AIHA的危险因素、发病机制、诊断及治疗方案研究进展作一综述,为移植后难治性/复发性AIHA的治疗提供更优策略。

In pediatric patients undergoing allogeneic hematopoietic stem cell transplantation(allo-HSCT),the incidence of autoimmune hemolytic anemia(AIHA)ranges from 2%to 6%.Risk factors include younger age at transplantation,non-malignant diseases,unrelated donor transplant,use of lymphocyte-depleting agents,and chronic graft-versus-host disease.These risk factors share the common characteristic of incomplete immune reconstitution or immune dysregulation post-HSCT,which may be related to the pathogenesis of AIHA.The treatment of post-transplant AIHA is challenging,with no standardized treatment guidelines currently available.Steroids remain the first-line treatment,but the relapse rate is high,with a complete remission rate of approximately 30%.Other conventional treatments such as intravenous immunoglobulin,plasma exchange,and splenectomy are usually ineffective for post-transplant AIHA.In recent years,some studies have explored second or third-line treatment options using monoclonal antibodies and immunosuppressive agents,with higher remission rates.However,the limited availability of studies makes sustained remission uncertain.This article reviews the progress in risk factors,pathogenesis,diagnosis and therapeutic options for post-transplant AIHA,providing improved strategies for the treatment of refractory/recurrent AIHA.