【摘要】 目的:研究抗人T细胞猪免疫球蛋白(anti-human T lymphocyte porcine immunoglobulin, p-ATG),环孢素A(Cyclosporin A,CsA)组成的强化免疫抑制治疗(intensive immunosuppressive therapy, IST)联合艾曲泊帕在重型再生障碍性贫血(severe aplastic anemia, SAA)患者的疗效及影响因素。方法:前瞻性登记纳入2020年4月—2022年8月25例初发SAA患者，接受p-ATG为基础的IST联合艾曲泊帕治疗。主要终点是6个月完全反应(complete response, CR)率和总体反应率(overall response rate, ORR)。次要终点是生存率、复发率及克隆演变风险。结果:治疗后6个月CR率和ORR分别为19.05%、71.43%。中位获得反应时间为3.00(0.10～11.80)个月，中位获得CR时间为14.40(0.90～20.00)个月，多因素分析显示疾病严重程度对获得反应时间有显著影响。中位随访时间11.30(0.70～26.40)个月，总生存率96.00%,中位无事件生存期为12.70(1.40～18.90)个月。1例于治疗后10个月出现克隆性造血。≥3级不良反应：感染8例，1例死亡；肝肾功能损害1例和牙周病2例。结论:p-ATG为基础的IST联合艾曲泊帕治疗SAA可获得较为满意的血液学反应率和反应速度，耐受性和安全性良好。更多还原

【Abstract】 Objective: To evaluate the efficacy and safety of anti-human T-cell porcine immunoglobin(p-ATG) based intensive immunosuppressive therapy(IST) combined with eltrombopag in patients with severe aplastic anemia(SAA). Methods: From April 2020 to August 2022, 25 newly-diagnosed SAA patients enrolled in a prospective cohort registry study were treated by p-ATG plus eltrombopag. The primary outcome was complete response(CR) and overall response rate(ORR) at 6 months. Secondary end points included survival, relapse, and clonal evolution. Results: CR and ORR at 6 months were 19.05% and 71.43%, respectively. The median time to first response was 3.00(0.10-11.80) months. The median time to achieve complete response was 14.40(0.90-20.00) months. Severity(vSAA vs SAA) was associated with the time to get first response. The median follow-up was 11.30(0.70-26.40) months, the over survival rate was 96.00%. The median event-free survival time was 12.70(1.40-18.90) months. Clonal hematopoiesis was found in one patient 10 months after initiation of treatment. Adverse events above grade 3 occurred in 8 cases of infection, including one death; 1 case of abnormal liver and renal functions, and 2 cases of parodontopathy, which were relieved by adjusting concentration of CsA. Conclusion: The addition of EPAG to p-ATG based IST was well tolerant and associated with high rates of hematologic response among previously untreated patients with SAA.更多还原