阿伐曲泊帕联合环孢素治疗输血依赖非重型再生障碍性贫血研究
Research of Avatrombopag combined with cyclosporine in the treatment of transfusion-dependent non-severe aplastic anemia
张婷 1徐丹 2陈小玉 3宫跃敏 3张雅文 3龙启强 4孙京男 5贾晋松 6何广胜 3师锦宁 1李建勇3
作者信息
- 1. 南京医科大学附属江宁医院血液内科,江苏南京 211199
- 2. 阜宁县人民医院血液科,江苏盐城 224499
- 3. 南京医科大学第一附属医院江苏省人民医院血液科,江苏南京 210029
- 4. 南京市第二医院南京中医药大学附属南京医院血液科,江苏南京 210037
- 5. 吉林大学第一医院血液肿瘤中心,吉林长春 130061
- 6. 北京大学人民医院血液科,北京 100044
- 折叠
摘要
目的 评估阿伐曲泊帕联合环孢素(Cyclosporin A,CsA)治疗在输血依赖型非重型再生障碍性贫血(transfusion-dependent non-severe aplastic anemia,TD-NSAA)患者中的疗效及安全性.方法 前瞻性登记分析从2020年10月到2023年10月,CsA联合阿伐曲泊帕治疗的21例TD-NSAA患者.结果 中位治疗时间7.5(1.0～27.0)个月,9例(42.86％)患者脱离输血,18例(85.71％)获得三系血液学反应(HR)[完全治疗反应(CR)3例(14.29％)、良好治疗反应(GPR)4例(19.05％)、部分治疗反应(PR)11例(52.38％)],无治疗反应(NR)3例(14.29％),中位起效时间1.6(0.1～7.4)个月,在第6、12个月分别17例(80.90％)、18例(85.71％)获得疗效.白细胞计数(2.21×109/L比3.82×109/L,P=0.029)、中性粒细胞计数(0.92×109/L 比 1.96×109/L,P=0.001)、血小板计数(13×109/L 比57×109/L,P=0.002)、网织红计数(1.84％比2.63％,P=0.011)均较治疗前显著升高.未发生与阿伐曲泊帕相关的2级或以上的不良事件.结论 阿伐曲泊帕联合CsA治疗TD-NSAA起效快,且安全性较好.
Abstract
Objective To evaluate the efficacy and safety of Avatrombopag treatment combined with Cyclosporin A(CsA)therapy in patients with transfusion-dependent non-Severe aplastic anaemia(TD-NSAA).Methods Prospective registration from October 2020 to October 2023,21 patients with TD-NSAA treated with CsA combined with Avatrombopag.Results The median duration of treatment with Avatrombopag was 7.5(1.0-27.0)months,and eighteen patients(85.71％)obtained trilineage hemalogical response:3 cases of complete treatment response(CR)(14.29％),4 case of good treatment response(GPR)(19.05％)and 11 cases of partial treatment response(PR)(52.38％),3 case of partial no treatment response(NR)(14.29％),9 patients(42.86％)achived transfusion dependent.17 cases(80.90％)and 18 cases(85.71％)achived remission at 6 months and 12 months,the median time to get response was 1.6 months(range 0.1-7.4 months).The white blood cell count(2.21 × 109/L vs.3.82 × 109/L,P=0.029),absolute neutrophil count(0.92×109/L vs.1.96×109/L,P=0.001),platelet count(13× 109/L vs.57× 109/L,P=0.002),and reticulocyte(1.84％vs.2.63％,P=0.011)improved markedly.No Avatrombopag-related grade 2 or higher adverse events occurred during treatment.Conclusion The combination of Avatrombopag and CsA might be an appropriate approach for TD-NSAA with good safety.
关键词
再生障碍性贫血/输血依赖/阿伐曲泊帕Key words
aplastic anaemia/transfusion-dependent/Avatrombopag引用本文复制引用
基金项目
国家自然科学基金(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(JNYYZXKY202214)
南京市江宁区科技惠民计划项目(2023081S)
出版年
2024
国家科技期刊平台
NSTL
万方数据