艾曲泊帕联合强化免疫抑制疗法治疗成人重型再生障碍性贫血疗效的预测因素

李瑞鑫; 金媛媛; 杨岩; 林圣云; 贾晋松; 孟凡凯; 张东华; 龙启强; 何广胜; 李建勇

doi:10.13201/j.issn.1004-2806.2022.05.007

艾曲泊帕联合强化免疫抑制疗法治疗成人重型再生障碍性贫血疗效的预测因素

- 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.
  林大学第一医院血液科
- 3.
  浙江省中医院血液科
- 4.
  北京大学人民医院血液科
- 5.
  华中科技大学同济医学院附属同济医院血液科
- 6.
  南京市第二医院血液科

详细信息

通讯作者: 何广胜，E-mail：heguangsheng1972@sina.com

中图分类号: R556.5

收稿日期: 2022-01-03

刊出日期: 2022-05-01

Predictors of eltrombopag combining with intensive immunosupressive therapy efficacy in adults with severe aplastic anemia

- 1.
  Department of Hematology, the First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Nanjing, 210029, China
- 2.
  Department of Hematology, the First Affiliated Hospital of Jilin University
- 3.
  Department of Hematology, Zhejiang Province Hospital of Traditional Chinese Medicine
- 4.
  Department of Hematology, Peking University People's Hospital, Peking University Institute of Hematology
- 5.
  Department of Hematology, Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology
- 6.
  Department of Hematology, the Second Hospital of Nanjing

More Information

Corresponding author: HE Guangsheng, E-mail: heguangsheng1972@sina.com

Received Date: 03 January 2022

Publish Date: 01 May 2022

摘要

摘要: 目的评价艾曲泊帕联合兔抗人胸腺细胞免疫球蛋白(ATG)和环孢素(CsA)强化免疫抑制疗法(IST)治疗中国成人重型再生障碍性贫血(SAA)的疗效及其预测因素。方法收集61例成人SAA患者接受艾曲泊帕联合IST治疗的临床资料，评估相关影响因素。结果 IST治疗后3、6和12个月，累计总有效率分别为67%、79%和85%，累计完全缓解率分别为21%、22%和32%。治疗前网织红细胞百分比(AUC=0.784，95%CI0.631~0.937，P=0.006)、红细胞分布宽度变异系数(AUC=0.737，95%CI0.527~0.947，P=0.022)、淋巴细胞绝对计数(AUC=0.733，95%CI0.563~0.903，P=0.025)或治疗前感染(AUC=0.705，95%CI0.519~0.891，P=0.048)能预测艾曲泊帕联合IST治疗的疗效。结论 IST前淋巴细胞绝对计数、网织红细胞百分比、红细胞分布宽度变异系数或治疗前感染能预测成人SAA患者对艾曲泊帕联合IST治疗的疗效。
- 重型再生障碍性贫血 /
- 艾曲泊帕 /
- 强化免疫抑制疗法 /
- 疗效
Abstract: Objective To evaluate the efficacy and predictors of eltrombopag combining with antithymocyte immunoglobulin(ATG) and cyclosporin A(CsA) in the treatment of severe aplastic anemia(SAA) in Chinese adults.Methods Clinical data of 61 adult SAA patients treated with eltrombopag combining with IST(ATG+CsA) were collected, and the related influencing fators were evaluated.Results The cumulative overall response rate of eltrombopag in combination with IST in the 3rd, 6th and 12th month were 67%, 79% and 85%, while the cumulative complete response rate in the 3rd, 6th and 12th month were 21%, 22% and 32%, respectively. The percentage of reticulocyte(AUC=0.784, 95%CI0.631-0.937,P=0.006), the red cell distribution width-coefficient of variation(AUC=0.737, 95%CI0.527-0.947,P=0.022), the absolute lymphocyte count(AUC=0.733, 95%CI0.563-0.903,P=0.025), or infection before treatment(AUC=0.705, 95%CI0.519-0.891,P=0.048) were correlated with the efficacy of eltrombopag in combination with IST.Conclusion The absolute lymphocyte count, the percentage of reticulocyte, the red cell distribution width-coefficient of variation before IST and infection before treatment can predict the efficacy of eltrombopag combined with IST in adult SAA patients.
- severe aplastic anemia /
- eltrombopag /
- intensive immunosupressive therapy /
- efficacy

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图 1 艾曲泊帕疗效预测的ROC曲线

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表 1 IST联合艾曲泊帕治疗6个月后有效和无效患者临床资料比较

临床资料	有效(45例)	无效(16例)	P
年龄/岁	50.5(24~74)	38(18~71)	0.073
性别/例			0.129
男	24	5
女	21	11
严重程度/例			0.020
SAA	34	7
VSAA	11	9
诊断至治疗时间/d	16(1~741)	27.5(3~1161)	0.720
红细胞计数/(×10¹²·L^-1)	1.9(0.9~4.1)	2.0(1.0~2.5)	0.770
血红蛋白/(g·L^-1)	61(37~128)	61.5(37~85)	0.800
血小板计数/(×10⁹·L^-1)	9(2~40)	7(4~19)	0.580
中性粒细胞计数/(×10⁹·L^-1)	0.4(0~4.0)	0.1(0~0.7)	0.019
淋巴细胞计数/(×10⁹·L^-1)	1.1(0.1~2.8)	0.8(0.1~2.0)	0.046
网织红细胞百分比/%	1.0(0.1~5.7)	0.4(0~1.5)	0.002
红细胞分布宽度变异系数/%	14.8(11.2~23.4)	12.6(10.8~19.9)	0.104
CD3/%	81.0(27.6~93.3)	83.6(60.0~89.9)	0.900
CD4/%	43.0(11.4~74.9)	50.8(34.8~67.5)	0.150
CD8/%	27.3(8.9~65.6)	23.3(14.8~41.0)	0.490
调节性T细胞/%	1.8(0~11.7)	2.7(0~9.2)	0.960
血清铁蛋白/(ng·mL^-1)	579.2(39.7~2 377.0)	1 101.7(146.9~5 597.5)	0.150
PNH克隆/例	8	2	0.920
治疗前感染/例	15	11	0.014

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表 2 IST治疗6个月后影响疗效的因素分析

影响因素	P
影响因素	单因素	多因素
年龄	0.051	0.071
严重程度(SAA vs VSAA)	0.024	0.540
诊断至治疗时间	0.400	0.290
治疗前感染(是vs否)	0.018	0.166
中性粒细胞计数	0.038	0.520
网织红细胞百分比	0.013	0.091

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