艾曲波帕治疗再生障碍性贫血

Eltrombopag for the treatment of aplastic anemia

再生障碍性贫血(aplastic anemia,AA)是一种骨髓造血衰竭综合征,是一种与骨髓细胞破坏和外周全血细胞减少相关的免疫介导性疾病[1-2],以骨髓造血功能低下、全血细胞减少和贫血/出血/感染征候群为主要的临床表现,其中出血和感染是重型再生障碍性贫血(severe aplastic anemia,SAA)引起死亡的主要原因。目前认为,T淋巴细胞异常活化以及功能亢进造成骨髓损伤,在原发获得性AA发病机制中占主要地位。

For many years,the focus in the clinical development of novel nontransplant therapies for aplastic anemia(AA)has been on intensifying immunosuppressive therapy,there are still some patients ineffective or relapse after immunosuppressive therapy.Eltrombopag is an oral thrombopoietin receptor agonist,which effectively improves the bone marrow function of aplastic anemia by stimulating the bone marrow.There are studies show that the rate of single drug treatment of AA about 40%to 50%,and there is a more significant improvement of blood cell counts by combining with immunosuppressive therapy.Up to now,the cumulative incidence of clonal evolution was not higher than that of AA treated with immunosuppressive therapy alone.More central clinical trials are needed to investigate the efficacy and safety of eltrombopag.