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原发免疫性血小板减少症住院患儿临床分析
引用本文:李蓉蔚,付荣凤,陈云飞,刘葳,薛峰,李慧媛,张磊,杨仁池,刘晓帆. 原发免疫性血小板减少症住院患儿临床分析[J]. 中国实验血液学杂志, 2021, 0(2): 574-580
作者姓名:李蓉蔚  付荣凤  陈云飞  刘葳  薛峰  李慧媛  张磊  杨仁池  刘晓帆
作者单位:中国医学科学院血液病医院(中国医学科学院血液学研究所)
摘    要:
目的:研究儿童原发免疫性血小板减少症(ITP)慢性化的影响因素,比较不同一线治疗方案的疗效。方法:回顾性分析2013年9月至2018年10月于本院住院治疗的ITP患儿的临床资料。结果:共计301例ITP患儿纳入研究。男150例,女151例,中位年龄8(0.17-17)岁。新诊断ITP 110例(36.5%),持续性ITP 92例(30.6%),慢性ITP 99例(32.9%)。中位随访时间41.92(1.07-74.03)个月。截至随访终点(2019年10月),202例新诊断/持续性ITP患儿中,79例(59例新诊断ITP,20例持续性ITP)在初诊后1年内获得缓解,缓解率39.3%;122例(50例新诊断ITP,72例持续性ITP)进展为慢性ITP,慢性化率60.7%;1例行脾切除。99例慢性ITP患儿中,5例行脾切除。多因素Logistic回归分析显示,隐匿起病(OR=3.754,95%CI:1.882-7.488,P=0.000)增加疾病慢性化风险,而血小板膜糖蛋白抗体阳性(OR=0.446,95%CI:0.224-0.888,P=0.021)降低慢性化风险,抗体类型的亚组分析无差异(P=0.305)。新诊断和持续性ITP仅接受一线治疗的患儿,单独应用静脉注射免疫球蛋白(IVIG)或IVIG联合激素疗效均优于激素单药治疗(P=0.028,0.028),而单独应用IVIG与IVIG联合激素比较疗效无差异(P=0.086)。结论:儿童ITP隐匿起病慢性化风险升高,血小板膜糖蛋白抗体阳性降低慢性化风险。儿童新诊断和持续性ITP的一线治疗,单独应用IVIG或IVIG联合激素疗效均优于激素单药治疗。

关 键 词:儿童  免疫性血小板减少症  慢性化  治疗  血小板膜糖蛋白抗体

Clinical Analysis of Hospitalized Children with Primary Immune Thrombocytopenia
LI Rong-Wei,FU Rong-Feng,CHEN Yun-Fei,LIU Wei,XUE Feng,LI Hui-Yuan,ZHANG Lei,YANG Ren-Chi,LIU Xiao-Fan. Clinical Analysis of Hospitalized Children with Primary Immune Thrombocytopenia[J]. Journal of experimental hematology, 2021, 0(2): 574-580
Authors:LI Rong-Wei  FU Rong-Feng  CHEN Yun-Fei  LIU Wei  XUE Feng  LI Hui-Yuan  ZHANG Lei  YANG Ren-Chi  LIU Xiao-Fan
Affiliation:(State Key Laboratory of Experimental Hematology,National Clinical Research Center for Blood Diseases,Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Tianjin Laboratory of Blood Disease Gene Therapy,CAMS Key Laboratory of Gene Therapy for Blood Diseases,Tianjin 300020,China)
Abstract:
Objective:To investigate the factors affecting the chronicity of childhood primary immune thrombocytopenia(ITP)and compare the efficiency of different first-line treatment regimens.Methods:Children with ITP hospitalized in our hospital from September 2013 to October 2018 were retrospectively analyzed.Results:Three hundred and one children(150 males and 151 females)were included in this study,with a median age of 8(0.17-17)years old,and 110(36.5%),92(30.6%),and 99(32.9%)cases were grouped into newly diagnosed,persistent,and chronic ITP,respectively.The median of follow-up was 41.92(1.07-74.03)months.At the end of the follow-up(October 2019),among the 202 newly diagnosed/persistent ITP children,79 cases(59 newly diagnosed and 20 persistent ITP)achieved remission within 1 year after initial diagnosis,with a remission rate of 39.3%;122 cases(50 newly diagnosed and 72 persistent ITP)developed chronic disease,with a chronicity rate of 60.7%;one case underwent splenectomy.In 99 cases with chronic ITP,5 cases underwent splenectomy.Multivariable logistic regression analysis showed that,the insidious onset of symptoms(OR=3.754,95%CI:1.882-7.488,P=0.000)increased the risk of chronicity,while the positive antibody to anti-platelet membrane glycoprotein(OR=0.446,95%CI:0.224-0.888,P=0.021)might reduce the risk of chronicity.And no difference was found by the analysis of subtype of anti-platelet membrane glycoprotein(P=0.305).The efficacy of the first-line treatment of intravenous immunoglobulin(IVIG)alone or combined with steroid was better than that of steroid alone(P=0.028,0.028),however,the efficiency was not significantly different between IVIG alone and combined with steroid(P=0.086).Conclusion:Insidious onset of symptoms in pediatric ITP increases the risk of chronicity,while the positive titer of anti-platelet membrane glycoprotein may reduce the risk.In the first-line treatment for the newly diagnosed/persistent children.The efficacy of IVIG alone or combined with steroid is better than that of steroid alone.
Keywords:children  immune thrombocytopenia  chronicity  treatment  anti-platelet membrane glycoprotein
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