艾曲泊帕联合强化免疫抑制疗法治疗成人重型再生障碍性贫血疗效的预测因素

Predictors of eltrombopag combining with intensive immunosupressive therapy efficacy in adults with severe aplastic anemia

目的:评价艾曲泊帕联合兔抗人胸腺细胞免疫球蛋白(ATG)和环孢素(CsA)强化免疫抑制疗法(IST)治疗中国成人重型再生障碍性贫血(SAA)的疗效及其预测因素。方法:收集61例成人SAA患者接受艾曲泊帕联合IST治疗的临床资料,评估相关影响因素。结果:IST治疗后3、6和12个月,累计总有效率分别为67%、79%和85%,累计完全缓解率分别为21%、22%和32%。治疗前网织红细胞百分比(AUC=0.784,95%CI 0.631~0.937,P=0.006)、红细胞分布宽度变异系数(AUC=0.737,95%CI 0.527~0.947,P=0.022)、淋巴细胞绝对计数(AUC=0.733,95%CI 0.563~0.903,P=0.025)或治疗前感染(AUC=0.705,95%CI 0.519~0.891,P=0.048)能预测艾曲泊帕联合IST治疗的疗效。结论:IST前淋巴细胞绝对计数、网织红细胞百分比、红细胞分布宽度变异系数或治疗前感染能预测成人SAA患者对艾曲泊帕联合IST治疗的疗效。

Objective:To evaluate the efficacy and predictors of eltrombopag combining with antithymocyte immunoglobulin(ATG)and cyclosporin A(CsA)in the treatment of severe aplastic anemia(SAA)in Chinese adults.Methods:Clinical data of 61 adult SAA patients treated with eltrombopag combining with IST(ATG+CsA)were collected,and the related influencing fators were evaluated.Results:The cumulative overall response rate of eltrombopag in combination with IST in the 3 rd,6 th and 12 th month were 67%,79%and 85%,while the cumulative complete response rate in the 3 rd,6 th and 12 th month were 21%,22%and 32%,respectively.The percentage of reticulocyte(AUC=0.784,95%CI 0.631-0.937,P=0.006),the red cell distribution width-coefficient of variation(AUC=0.737,95%CI 0.527-0.947,P=0.022),the absolute lymphocyte count(AUC=0.733,95%CI 0.563-0.903,P=0.025),or infection before treatment(AUC=0.705,95%CI 0.519-0.891,P=0.048)were correlated with the efficacy of eltrombopag in combination with IST.Conclusion:The absolute lymphocyte count,the percentage of reticulocyte,the red cell distribution width-coefficient of variation before IST and infection before treatment can predict the efficacy of eltrombopag combined with IST in adult SAA patients.