艾曲泊帕联合环孢素A治疗输血依赖非重型再生障碍性贫血疗效分析

The efficacy of eltrombopag plus cyclosporine A in patients with transfusion-dependent non-severe aplastic anemia:a retrospective study from single center

回顾性分析2019年至2021年于江苏省人民医院血液科接受艾曲泊帕联合环孢素A(CsA)的13例初治输血依赖非重型再生障碍性贫血(transfusion-dependent non-severe aplastic anemia,TD-NSAA)患者的临床资料,评估艾曲泊帕联合CsA治疗TD-NSAA的疗效、不良反应等。13例TD-NSAA患者治疗3个月总体血液学反应(OR)12/13。至随访截点,12例患者获得治疗反应,其中2例达完全血液学反应(CR),9例达部分血液学反应(PR),1例达血液学反应(HR)。1例患者在艾曲泊帕治疗6个月时转为阵发性睡眠性血红蛋白尿。5例患者治疗期间出现不同程度的不良反应,但未对治疗造成影响。对比既往单一CsA方案疗效,对于TD-NSAA,艾曲泊帕联合CsA应用可提高疗效且安全性较好。

The main purpose of our study was to evaluate the efficacy and safety of eltrombopag plus cyclosporine A(CsA)in transfusion-dependent non-severe aplastic anemia(TD-NSAA).The clinical characteristics of 13 TD-NSAA patients who received initial treatment of eltrombopag plus CsA from 2019 to 2021 were retrospectively analyzed.The 3-month overall hematological response(OR)rate was 12/13.Until the end of follow-up,12 patients responded,among whom 2 patients reached complete response(CR)and 9 patients reached partial response(PR)and 1 with HR.Paroxysmal nocturnal hemoglobinuria(PNH)developed in one patient at 6 months after treatment.Five of thirteen patients reported mild adverse reactions,which were all manageable.Compared with historical data,the combination of eltrombopag with CsA is an effective regimen in patients with TD-NSAA.