获得性纯红细胞再生障碍患者治疗的研究现状  

Research status on treatment of acquired pure red cell aplasia patients

作　　者：刘鑫垚 龙章彪 Liu Xinyao;Long Zhangbiao(Department of Hematology,First Affiliated Hospital of Anhui Medical University,Hefei 230032,Anhui Province,China)

机构地区：[1]安徽医科大学第一附属医院血液内科,合肥230032

出　　处：《国际输血及血液学杂志》2023年第4期300-306,共7页International Journal of Blood Transfusion and Hematology

基　　金：国家自然科学基金(81900118)。

摘　　要：纯红细胞再生障碍(PRCA)是一种罕见的红系造血衰竭所致贫血性疾病。该病按照病因可被分为先天性PRCA和获得性PRCA,后者病程长、易复发。环孢素A、环磷酰胺与糖皮质激素等一线治疗对部分获得性PRCA患者有效,但仍有部分复发/难治性患者预后差,对一线治疗方案无反应。近年来涌现出部分新型免疫抑制药物、靶向治疗等方法,为复发/难治性患者带来希望。笔者拟就获得性PRCA的一般支持、新型免疫抑制剂、单克隆抗体治疗,造血干细胞移植(HSCT),以及特殊类型继发性获得性PRCA的治疗等方面,对获得性PRCA治疗的研究现状进行阐述,旨在为获得性PRCA患者的治疗提供参考。Pure red cell aplasia(PRCA)is a rare anemia disease caused by erythroid hematopoietic failure.According to the etiology,the disease can be divided into congenital PRCA and acquired PRCA.The latter has a long course of disease and is prone to relapse.The first-line treatment,such as cyclosporine A,cyclophosphamide and glucocorticoid are effective for some patients with acquired PRCA,but there are some relapsed/refractory patients with poor prognosis and no respond to previous first-line treatment.In recent years,there are some new immunosuppressive drugs and targeted therapy,which bring hope to relapsed/refractory patients.In order to provide reference for treatment of acquired PRCA patients,this article elaborate on research status in treatment of acquired PRCA in aspects of general support,new immunosuppressive agents,monoclonal antibody,hematopoietic stem cell transplantation(HSCT),and treatment of specific types of secondary acquired PRCA.

关 键 词：红细胞再生障碍 纯 免疫抑制剂 西罗莫司 抗体 单克隆 造血干细胞移植 

分 类 号：R556.5[医药卫生—血液循环系统疾病]