中华风湿病学杂志2024,Vol.28Issue(7) :465-471.DOI:10.3760/cma.j.cn141217-20230410-00090

艾曲波帕治疗结缔组织病继发免疫性血小板减少的疗效分析

Clinical efficacy of eltrombopag in the treatment of autoimmune thrombocytopenia secondary to con-nective tissue diseases

任田 常新 曾克勤 周欣 周二叶 武剑
中华风湿病学杂志2024,Vol.28Issue(7) :465-471.DOI:10.3760/cma.j.cn141217-20230410-00090

艾曲波帕治疗结缔组织病继发免疫性血小板减少的疗效分析

Clinical efficacy of eltrombopag in the treatment of autoimmune thrombocytopenia secondary to con-nective tissue diseases

任田 1常新 1曾克勤 1周欣 1周二叶 1武剑1
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作者信息

  • 1. 苏州大学附属第一医院风湿免疫科,苏州 215006
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摘要

目的 探讨艾曲波帕治疗结缔组织病继发免疫性血小板减少的疗效和安全性.方法 回顾性收集2020年1月至2022年12月在苏州大学附属第一医院风湿免疫科应用艾曲波帕治疗的结缔组织病继发血小板减少患者共23例作为治疗组,并收集同期未应用艾曲波帕的34例患者作为对照组,观察治疗前及随访时血小板计数、应答情况、不良反应等.采用SPSS 26.0软件进行统计学分析.计量资料的组间比较采用独立样本t检验或秩和检验.计数资料组间比较采用x2检验或Fisher确切概率法.结果 艾曲波帕组患者治疗后最高总有效率出现在第12周为82.6%(19/23).治疗1周[(47±29)×109/L,t=-5.25,P<0.001]、治疗 2周[(111±87)×109/L,t=-5.31,P<0.001]、治疗 4 周[(150±104)×109/L,t=-6.23,P<0.001]、治疗 12 周[(153±92)×109/L,t=-6.64,P<0.001]、治疗 24 周[(134±83)×109/L,t=-7.11,P<0.001]时血小板计数均较基线[(14±8)×109/L]明显上升且差异均有统计学意义.与常规治疗组比较,艾曲波帕组患者既往使用免疫球蛋白(IVIG)患者比例更高[60.9%(14/23)与20.6%(7/34),x2=9.57,P=0.002],现合并使用IVIG患者比例更低[21.7%(5/23)与61.8%(21/34),x2=8.86,P=0.003],而激素使用最大日剂量更低[80(50,120)mg/d与80(55,115)mg/d,Z=-2.02,P=0.042],且差异均有统计学意义.随访24周,服药期间所有患者均未发生与艾曲波帕相关严重不良反应.结论 艾曲波帕治疗结缔组织病继发免疫性血小板减少疗效显著,安全性高,可以作为常规治疗效果不佳或不宜应用大剂量激素患者的选择之一.

Abstract

Objective To explore the efficacy and safety of eltrombopag in the treatment of immune thrombocytopenia secondary to connective tissue diseases.Methods Totally 23 patients with immune associated thrombocytopenia secondary to connective tissue diseases who were treated with eltrombopag in the Department of Rheumatology,the First Affiliated Hospital of Soochow University,from January 2020 to December 2022 were selected as the treatment group.Additionally,34 patients who did not receive eltrombopag during the same period were collected as the control group.Statistical analysis was conducted using SPSS 26.0 software.The t test or Wilcoxon rank-sum test were used to compare differences between groups of categorical data.The chi-square test or Fisher's exact test was used to compare the differences between groups of quantitative data.Results The highest overall response rate of the 23 patients was 82.6%(19/23),which occurred at the 12th week after treatment.The blood platelet count was significantly increased from baseline at the first week[(47±29)×109/L,t=-5.25,P<0.001],also at the 2nd week[(111±87)×109/L,t=-5.31,P<0.001],the 4th week[(150±104)×109/L,t=-6.23,P<0.001],the 12th week[(153±92)×109/L,t=-6.64,P<0.001],the 24th week[(134±83)×109/L,t=-7.11,P<0.001],and the difference was statistically significant.Compared with the conventional treatment group,patients in the eltrombopag group had a higher proportion of patients previously being treated with IVIG[60.9%(14/23)vs.20.6%(7/34),x2=9.57,P=0.002],a lower proportion of patients currently using IVIG[21.7%(5/23)vs.61.8%(21/34),x2=8.86,P=0.003],and a lower maximum daily dose of glucocorticoid use[80(50,120)mg/d vs.80(55,115)mg/d,Z=-2.02,P=0.042],the differences were statistically significant.After being followed up for 24 weeks,no patients experienced any serious adverse reactions related to eltrombopag.Conclusion Eltrombopag is effective and safe in the treatment of immune thrombocytopenia secondary to connective tissue diseases.It can be used as one of the choices for patients who failed to respond to conventional treatment or patients who could not tolerate high-dose glucocorticoids.

关键词

结缔组织疾病/紫癜,血小板减少性/艾曲波帕

Key words

Connective tissue diseases/Purpura,thrombocytopenic/Eltrombopag

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出版年

2024
中华风湿病学杂志
中华医学会

中华风湿病学杂志

CSTPCD
影响因子:0.651
ISSN:1007-7480
参考文献量2
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