重组人血小板生成素治疗儿童原发性免疫性血小板减少症的有效性和安全性:系统综述与meta分析

Efficacy and safety of recombinant human thrombopoietin in the treatment of primary immune thrombocytopenia in children:A systematic review and meta-analysis

目的评价重组人血小板生成素(rh TPO)治疗儿童原发性免疫性血小板减少症(ITP)的有效性和安全性。方法采用主题词和自由词结合的方式在Embase、Pubmed、CENTRAL、万方数据知识服务平台(Wanfang)、维普中文科技期刊数据库(VIP)、中国知网(CNKI)系统检索建库至2020年6月24日间rhTPO治疗儿童ITP的随机对照试验与非随机对照试验,按照既定的纳入排除标准筛选合格研究,并采用Cochrane工具和ROBINS-I工具评估文献的偏倚风险,由2名研究者独立提取相关数据,之后采用R3.5.2进行meta分析。结果共有8项研究纳入分析,其中随机对照试验5篇,非随机对照试验3篇,总计786例患者。各项研究均报告治疗有效率,其中2项研究试验组单独采用rhTPO,合并治疗有效率为93.46%;3项研究的试验组采用rhTPO联合糖皮质激素,随机效应模型合并后治疗有效率为92.11%。试验组共报告不良反应患者8例(5.26%),对照组为22例(14.57%),异质性检验显示差异有统计学意义(P=0.86,I2=0),采用固定效应模型合并的RR值为0.39(95%CI:0.18~0.81)。结论单独使用rhTPO或联合使用糖皮质激素治疗儿童ITP的有效性优于单独使用糖皮质激素,但现有研究的质量不高,需要开展多中心随机双盲安慰对照试验作为更有力的证据支撑。

Objective To analyze the efficacy and safety of recombinant human thrombopoietin(rhTPO)in the treatment of primary immune thrombocytopenia(ITP)in children.Methods The randomized controlled trials and non-randomized controlled trial of children with ITP treated by rhTPO were searched for in Embase,Pubmed CENTRAL,Wanfang,VIP,and CNKI until June 24,2020. The Cochrane and ROBINS-I tools were used to assess the bias risk of literature. Qualified studies were screened according to the established inclusion and exclusion criteria,and then data was extracted by two researchers.The meta-analysis was proceeded by R3.5.2.Results A total of 8 studies,including 5 randomized controlled trials and 3 non-randomized controlled trials were included in analysis with a total of 786 patients. All the studies reported treatment response rate,among which the two experimental groups used rhTPO separately,and the combined response rate was 93.46%.Three studies used rhTPO combined with glucocorticoids in experimental groups,and the effective rate was 92.11%,calculated by random effect model. A total of 8 patients(5.26%)in the experimental group and 22 patients(14.57%)in the control group who reported adverse reactions. Heterogeneity test showed that the difference was statistically significant(P=0.86,I2=0),and the RR value of the fixed-effect model was 0.39(95%CI:0.18~0.81).Conclusion The efficacy of using rhTPO alone or in combination with glucocorticoid to treat ITP in children is better than that of using glucocorticoid alone. However,the quality of the existing studies is not high,a multi-center randomized double-blind placebo controlled trial is needed to provide stronger evidence support.