免疫抑制治疗再生障碍性贫血疗效预测指标研究进展

Progress of predicting response to immunosuppressive therapy in aplastic anemia

再生障碍性贫血（AA）是一类以骨髓造血功能衰竭和全血细胞减少为特征的严重血液病。目前对缺乏全相合同胞供者的AA患者，多采用抗胸腺细胞球蛋白（ATG）联合环孢素的免疫抑制治疗（IST），约1/3的患者IST无效，且IST存在一定临床风险，严重不良反应可致ATG治疗相关死亡。因此，有必要开展有关IST的疗效预测研究，为临床提供较为明确的疗效预测指标，使IST病例选择更为合理，从而进一步提高远期疗效。现就近年来国内外IST疗效预测的相关研究进行综述，以期为临床IST提供参考，并为后续相关研究提供依据。

Aplastic anemia is a severe hematological disease characterized by bone marrow failure and pancyto- penia. Immunosuppressive therapy（IST） with antithymocyte globulin（ATG） and cyclosporine A（CSA） is the first -line treatment for patients who lack an HLA - matched sibling. One third of the patients has no response to IST and the high risk of ATG - related adverse effects may lead to ATG - related death. So it is necessary to explore the predictive marker of response to IST. A defined predictive marker may make the selection of treatment more reasonable and further improve the long - term efficacy. The relevant literatures were analyzed and summarized in recent years, in order to pro- vide a reference for clinical treatment and a baseline for prospective studies.