慢性免疫性血小板减少症患儿免疫抑制基础上促血小板生成素类药物的疗效研究  

TPO-RAs based on immunosuppression can improve the curative effect of chronic immune thrombocytopenia in children

作　　者：张佳璐 傅玲玲[1] 陈振萍 谢幸娟 朱筱旌 马洁[1] 姚佳峰[1] 张利强[1] 吴润晖[1] ZHANG Jialu;FU Lingling;CHEN Zhenping;XIE Xingjuan;ZHU Xiaojing;MA Jie;YAO Jiafeng;ZHANG Liqiang;WU Runhui(National Children′s Medical Center,Beijing Children′s Hospital Affiliated to Capital Medical University,Blood Disease Center,Beijing Key Laboratory of Children′s Blood Disease and Tumor Molecular Typing,National Key Discipline of Pediatrics,Key Laboratory of the Ministry of Education for Research on Major Pediatric Diseases,Beijing,100045 China;National Children′s Medical Center,Beijing Children′s Hospital Affiliated to Capital Medical University,Blood Disease Center,Beijing Institute of Pediatrics,Blood Disease Laboratory,Beijing 100045,China)

机构地区：[1]国家儿童医学中心,首都医科大学附属北京儿童医院,血液病中心,儿童血液病与肿瘤分子分型北京市重点实验室,儿科学国家重点学科,儿科重大疾病研究教育部重点实验室,北京100045 [2]北京市儿科研究所,血液疾病研究室

出　　处：《中国小儿血液与肿瘤杂志》2023年第3期148-152,共5页Journal of China Pediatric Blood and Cancer

基　　金：北京市医院管理中心儿科学科协同发展中心“儿科专项”(项目编号:XTZD20180205);国家自然科学基金课题(项目编号:81970111);北京市属医院科研培育计划(项目编号:PX2022051);北京市属医院科研培育计划(项目编号:PX20231202)。

摘　　要：目的旨在比较序贯大剂量地塞米松、利妥昔单抗免疫抑制治疗后加用艾曲波帕与直接应用艾曲波帕治疗的儿童慢性免疫性血小板减少症(CITP)数据,证实免疫抑制基础上的促血小板生成治疗可提高儿童CITP疗效。方法本研究为单中心非随机对照研究,将2018年1月—2021年8月入组的CITP患儿分为两组:(1)直接应用艾曲波帕(简称直接组);(2)经大剂量地塞米松、利妥昔单抗升阶梯治疗无效时加用艾曲治疗(简称序贯组)。评价两组疗效、安全性和药物经济学指标。结果共入组48例患儿,男性23例,女性25例,中位发病年龄为5.0(3.6-7.5)岁,病程均超过一年,治疗前血小板(PLT)计数为10.0(4.0-15.0)×10^(9)/L。(1)基线数据:直接组男16例,女13例,序贯组男7例,女12例。两组的发病年龄、病程、PLT计数等基线无统计学差异(P>0.05)。(2)疗效:直接组的总体反应率为69%,完全反应率21%,无效率31%;序贯组分别为84%、26%和16%。直接组艾曲波帕平均治疗时长为8.2个月,停药率41%,62%的患儿PLT水平维持在50×10^(9)/L以上水平,平均治疗费用为7.38万元;序贯组艾曲波帕平均治疗时长为7.0个月,停药率58%,74%的患儿PLT维持在50×10^(9)/L以上,平均治疗费用为9.87万元;停药率及PLT维持在50×10^(9)/L以上的患儿比例,序贯组均高于直接组,但不存在统计学差异。(3)安全性:直接组药物相关不良事件3例(10%),序贯组8例(42%),无统计学差异(P>0.05)。结论与直接应用艾曲波帕治疗儿童CITP相比,在免疫抑制剂治疗基础上应用艾曲波帕,疗效更好且不增加副作用和经济负担,可考虑作为儿童CITP二线治疗的优选策略。Objective The purpose of our study is to compare the efficacy of eltrombopag therapy alone and eltrombopag therapy after high-dose dexamethasone and rituximab treatment ineffective,confirming the feasibility of escalating the treatment strategy.Methods This was a non-randomized,single-center clinical study.Data from children with CITP in our center were collected between January 2018 and August 2021.They were divided into two groups according to the second-line treatment:the monotherapy group(eltrombopag)and the combination group(escalating treatment strategy).Therapeutic effect were evaluated after 12 months of follow-up.Results A total of 48 cases(23 males and 25 females)were included.The median age was 5.0(3.6-7.5)years old.The median platelet count was 10.0(4.0-15.0)×109/L before treatment.There was no difference between the two groups at baseline(P>0.05).The combination group resulted in a higher incidence of response(84%vs 69%,P>0.05)and complete response(26%vs 17%,P>0.05)compared with the monotherapy group.There was no difference in the mean treatment duration,drug withdrawal rate,and cost between the two groups(P>0.05).No serious adverse events were reported.Conclusions This study suggests that escalating treatment strategy can achieve better efficacy than TPO-RAs without increasing the cost of treatment.It is an effective,safe and second-line treatment scheme suitable for Chinese patients.

关 键 词：儿童 免疫性血小板减少症 艾曲波帕 免疫抑制剂 

分 类 号：R725.5[医药卫生—儿科]