再生障碍性贫血的治疗进展

Recent progresses in the treatment of aplastic anemia

重型再生障碍性贫血（SAA）是威胁生命的严重疾病,病死率高,随着造血干细胞移植等治疗的开展,这部分患者生存率已得到极大的提高.SAA一旦诊断需立即开始治疗.对于非输血依赖的非SAA患者,可选择观察和支持治疗.年轻（小于40岁）有同胞全相合供者的SAA患者首选造血干细胞移植,没有条件的患者首选免疫抑制治疗.免疫抑制治疗国外一线推荐马抗胸腺细胞球蛋白（ATG）;国内相关研究表明兔ATG和猪抗淋巴细胞免疫球蛋白（ALG）都可作为一线治疗,血液学缓解率可达60％～70％.复发、难治的SAA患者,可考虑无关供者骨髓移植、第2次ATG治疗或阿伦单抗治疗.间充质干细胞治疗、促血小板生成素受体（c-MPL）拮抗剂艾曲波帕及左旋咪唑在难治性及复发的SAA患者中也表现出了一定的疗效,期待进一步的临床研究.

Severe aplastic anemia（SAA） is a life-threatening disease,with a high fatality rate.New treatments such as bone marrow transplantation（BMT）,immunosuppressive therapy and supportive care have greatly improved the survival of patients with SAA.For the patients with non-SAA,especially for those who are transfusionindependent,observation or supportive care is often sufficient.However,for the SAA patients younger than 40 years and having HLA-identical sibling donor,allogenic bone marrow transplantation is recommended as an initial choice.Immunosuppressive therapy is the front-line choice for the SAA patients older than 40 years and having no HLA-identical sibling donor or the non-SAA patients with transfusion-dependency.The standard immunosuppressive regimen is a combination of antithymocyte globulin（ATG） and cyclosporine.According to the researches from Europe and America,horse ATG is recommended for the first line immunosuppressive therapy（IST）.But several studies in China have revealed that rabbit ATG and pig antilymphocyte globulin（ALG） can also be very effective,with a hematological recovery rate up to 60％ to 70％.For the patients with one course of IST failed or the patients with refractory or relapsed SAA,matched unrelated donor BMT,second course of IST or alemtuzumab may be considered.In addition,other treatments such as mesenchymal stem cell transplantation,eltrombopag and levamisole,also show benefits in the treatment of refractory and relapsed SAA.