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

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 TPO-RAs also improved the response rates of TPO-RAs.