重型再生障碍性贫血的非移植治疗:第58届美国血液学会年会报道  

Non-transplant therapy for severe aplastic anemia

作　　者：刘为易 胡晓梅[1] 

机构地区：[1]中国中医科学院西苑医院血液科,北京100091

出　　处：《临床血液学杂志》2017年第6期829-833,共5页Journal of Clinical Hematology

摘　　要：在过去的3年间,再生障碍性贫血（aplastic anemia,AA）患者通过异基因造血干细胞移植（HSCT）或马抗胸腺细胞球蛋白（hATG）和环孢素（CSA）的强化免疫抑制治疗（IST）,获得了有效的造血恢复。虽然供体造血干细胞的成功植入是AA唯一明确有效的治疗,但因匹配供者的缺乏、与老龄和并发症有关的HSCT的异常毒性、过长时间等待合适的供者,许多患者无法进行HSCT。大部分接受IST的患者预后良好,此方法彻底改变了AA患者的结果。Non-transplant therapeutic options for acquired and constitutional aplastic anemia have significantly expanded during the last 5years.In the future,transplant may be required less frequently.That trilineage hematologic responses could be achieved with the single agent eltrombopag in refractory aplastic anemia promotes new interest in growth factors after years of failed trials using other growth factor agents.Preliminary results adding eltrombopag to immunosuppressive therapy are promising,but long-term follow-up data evaluating clonal evolution rates are required before promoting its standard use in treatment-naive disease.Danazol,which is traditionally less preferred for treating cytopenias,is capable of preventing telomere attrition associated with hematologic responses in constitutional bone marrow failure resulted from telomere disease.Non-transplant therapy for bone marrow failure will be summarized in this paper based on the reports in the 58th ASH annual meeting.

关 键 词：骨髓衰竭 再生障碍性贫血 血小板生成素受体 雄激素 端粒 

分 类 号：R556.5[医药卫生—血液循环系统疾病]