抗人T细胞猪免疫球蛋白联合艾曲泊帕治疗初发重型再生障碍性贫血疗效分析  

Anti-human T-cell porcine immunoglobin combined with eltrombopag in patients with severe aplastic anemia

作　　者：宫跃敏 马永超 陈小玉 李瑞鑫 李悦[1] 张雅文[1] 师锦宁 龙启强[3] 杨岩[4] 贾晋松[5] 林圣云[6] 何广胜[1] 李建勇[1] GONG Yuemin;MA Yongchao;CHEN Xiaoyu;LI Ruicin;LI Yue;ZHANG Yawen;SHI Jinning;LONG Qiqiang;YANG Yan;JIA Jinsong;LIN Shengyun;HE Guangsheng;LI Jianyong(Department of Hematology,the First Affiliated Hospital of Nanjing Medical University,Jiangsu Province Hospital,Nanjing,210029,China;Department of Hematology,the Affiliated Jiangning Hospital of Nanjing Medical University;Department of Hematology,Second Hospital of Nanjing,the Affiliated Nanjing Hospital of Nanjing Traditional Chinese Medical University;The Hematology&Tumor Center of the First Hospital of Jilin University;Department of Hematology,People's Hospital of Peking University;Department of Hematology,Zhejiang Traditional Chinese Medicine Hospital)

机构地区：[1]南京医科大学第一附属医院、江苏省人民医院血液科,南京210029 [2]南京医科大学附属江宁医院血液内科 [3]南京市第二医院南京中医药大学附属南京医院血液科 [4]吉林大学第一医院血液肿瘤中心 [5]北京大学人民医院血液科 [6]浙江省中医院血液科

出　　处：《临床血液学杂志》2023年第7期482-487,共6页Journal of Clinical Hematology

基　　金：国家自然科学基金(No:81900109);南京医科大学附属江宁医院免疫细胞转化研究中心开放课题(No:JNYYZXKY202214)。

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。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.

关 键 词：重型再生障碍性贫血 抗人T细胞免疫球蛋白 艾曲泊帕 

分 类 号：R556.5[医药卫生—血液循环系统疾病]