艾曲波帕联合环孢素A治疗重型再生障碍性贫血疗效及安全性观察

Efficacy and safety of eltrombopag combined with cyclosporine A in the treatment of severe aplastic anemia

目的 探讨艾曲波帕联合环孢素A(CsA)治疗重型再生障碍性贫血(SAA)的疗效及安全性。方法 回顾性分析2018年1月至2021年8月苏州大学附属第一医院68例接受艾曲波帕联合CsA治疗的SAA患者资料,观察治疗后的疗效、并发症及转归。结果 治疗后3个月、6个月的总有效率(ORR)分别为32.4%和42.6%。ORR与艾曲波帕使用时间长短有明显相关性(P<0.001)。中位随访时间27.5(6.0~47.0)个月,2年总生存率(OS)为(87.7±4.1)%,OS与从诊断至艾曲波帕开始使用间隔长短有显著相关性(P=0.010)。2年无失败生存率(FFS)为(42.6±6.0)%,年龄(P=0.040)、治疗前网织红细胞百分比(P<0.001)及艾曲波帕使用时间长短(P<0.001)与FFS有显著相关性。治疗过程中不良反应主要表现为胆红素升高,发生率为77.9%,均较轻微,经适当处理后有效控制。8例患者(11.8%)死亡,其中肺部感染5例(7.4%),消化道出血1例(1.5%),脑出血2例(2.9%)。结论 艾曲波帕联合CsA治疗SAA安全有效,患者耐受良好,无严重不良反应,值得进一步探索。

Objective To explore the efficacy and safety of eltrombopag combined with cyclosporine A(CsA)in treatment of severe aplastic anemia(SAA)patients.Methods We retrospectively analyzed clinical statistics of 68 SAA patients treated with eltrombopag combined with CsA in the First Affiliated Hospital of Soochow University from January 2018 to August2021.The primary outcome was complete hematologic response at 3 months and 6 months.Secondary end points included overall response,failure free survival,adverse events.Results The overall response rates at 3 months and 6 months were32.4%,42.6%,respectively.Patients with longer eltrombopag use displayed higher ORR(P<0.001).The median follow-up time was 27.5(6.0-47.0)months.At a median follow-up of 2 years,the overall survival rate(OS) was(87.7±4.1)%.Patients who experienced short interval between diagnosis and treatment displayed higher OS rates(P=0.010).The failure free survival(FFS)was(42.6 ± 6.0)%.Age(P=0.040),percentage of reticulocytes before treatment(P<0.001)and the duration of eltrombopag use(P<0.001)were statistically significant correlated with FFS.Slight increase of bilirubin was the main adverse reaction.The incidence was 77.9%.It can be effectively controlled after appropriate treatment.A total of 8patients(11.8%)died,including 5 cases(7.4%)died of pulmonary infection,1 case(1.5%)died of gastrointestinal bleeding and 2 cases(2.9%)died of intracerebral hemorrhage.Conclusions The use of eltrombopag combined with Cs A in SAA is effective.The patients tolerated it well,and without causing severe adverse effect.It is worthy of further exploration of this regimen.