升阶梯方案提高儿童慢性免疫性血小板减少症疗效——单中心数据分析  

The escalating treatment strategy improve the efficacy of chronic immune thrombocytopenia in children from a single-center

作　　者：胡玉 马静瑶[1] 张佳璐 谢幸娟 刘会青 谷昊[1] 傅玲玲[1] 吴润晖[1] HU Yu;MA Jingyao;ZHANG Jialu;XIE Xingjuan;LIU Huiqing;GU Hao;FU Lingling;WU Runhui(Hematology Center,Beijing Key Laboratory of Pediatric Hematology Oncology,National Key Discipline of Pediatrics(Capital Medical University),Key Laboratory of Major Diseases in Children,Ministry of Education,Beijing Children′s Hospital,Capital Medical University,National Center for Children′s Health,China,100045)

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

出　　处：《中国小儿血液与肿瘤杂志》2023年第1期26-31,共6页Journal of China Pediatric Blood and Cancer

基　　金：国家自然科学基金课题(项目编号:81970111);北京市自然科学基金课题(项目编号:7192064);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);国家科技重大专项(项目编号:2017ZX09304029001)。

摘　　要：目的探讨升阶梯方案提高儿童慢性免疫性血小板减少症(CITP)的疗效。方法采用单中心、前瞻性队列研究方法,收集2018年6月—2020年6月于我院应用升阶梯方案治疗的儿童CITP临床数据。升阶梯方案包括阶梯Ⅰ:4~6轮大剂量地塞米松;阶梯Ⅱ:小剂量利妥昔单抗;阶梯Ⅲ:艾曲波帕。根据最终是否依从升阶梯方案治疗分为依从组和未依从组,开始治疗1年为终点进行疗效评价。结果共纳入63例CITP患儿,中位随访时间30(12~42)个月,其中依从组33例,未依从组30例。治疗1年后,依从组总反应率(TRR)94%,未依从组为33%。阶梯Ⅰ使30%(TRR 13/43)患儿获得疗效;而序贯在免疫抑制治疗后的艾曲波帕单药治疗(阶梯Ⅲ)可以提高促血小板受体激动剂(TPO-RAs)疗效(TRR 82%,9/11例),高于不依从组中直接应用艾曲波帕病例TRR(75%,3/4例)。两组病例不良事件比较无差异(P>0.05)。结论升阶梯方案提高儿童CITP疗效。短疗程糖皮质激素反复冲击治疗可以使部分患儿获益;在未增加副作用的情况下,短疗程糖皮质激素无效病例从糖皮质激素、利妥昔单抗序贯到TPO-RAs的升阶梯治疗方式也提高了TPO-RAs的有效率。Objective The purpose of this study was to investigate the effect of the escalating treatment strategy on improving the efficacy of CITP in children.Methods A single-center,prospective cohort study was conducted.Clinical data of children CITP with the escalating treatment strategy in our hospital were collected from June 2018 to June 2020.The escalating strategy included three steps:Step I(six courses of high-dose dexamethasone[HDD]),StepⅡ(HDD combined with low-dose rituximab),and StepⅢ(eltrombopag).The patients were divided into compliant and non-compliant groups according to whether they finally complied with the escalating treatment strategy,and the efficacy was evaluated and analysed one year after the treatment.Results A total of 63 children with CITP were included,after a median follow-up of 30(12-42)months,the enrolled children were divided into the compliance group(33 cases)and the non-compliance group(30 cases),and there was no difference in baseline characteristics between the two groups.After 1 year of treatment,TRR of the compliance group was 94%,and TRR of the non-compliance group was 33%.The efficacy of the compliance group was significantly higher than that of the non-compliance group,and higher effective rate was maintained during continuous observation.High-dose dexamethasone(step I in the compliance group)achieved efficacy in 30%of children(TRR 13/33).Sequential eltrombopag after immunosuppression(stepⅢin the compliance group)improved TPO-RAs efficacy(TRR 82%,9/11)compared with eltrombopag monotherapy in the non-compliance group(TRR 75%,3/4).There was no difference in adverse events between 2 groups(P>0.05).Conclusions The escalating treatment strategy can improve the effect of CITP in children.The application of a short course of high-dose dexamethasone can make some children benefit and avoid the subsequent treatment.Without increased side effects,for patients who failed to the short course of high-dose dexamethasone,the escalating treatment therapy from glucocorticoid and rituximab to T

关 键 词：慢性免疫性血小板减少症 儿童 治疗 疗效 

分 类 号：R725.5[医药卫生—儿科]