儿童激素耐药肾病综合征应用利妥昔单抗疗效及安全性的系统评价和Meta分析  

Efficacy and safety of rituximab in children with steroid-resistant nephrotic syndrome:A systematic review and meta-analysis

作　　者：曾月桂 徐可[1] 王芳[1] ZENG Yuegui;XU Ke;WANG Fang(Department of Pediatrics,Children's Medical Center,Peking University First Hospital,Beijing 100034,China;Department of Pediatric Hematology Oncology,Rheumatology and Nephrology,Zhangzhou Affiliated Hospital of Fujian Medical University/Zhangzhou Municipal Hospital of Fujian Province,Zhangzhou 363000,China)

机构地区：[1]北京大学第一医院儿童医学中心儿内科,北京100034 [2]福建医科大学附属漳州市医院/福建省漳州市医院儿童血液风湿肾科,漳州363000

出　　处：《中国循证儿科杂志》2025年第2期124-130,I0025-I0040,共23页Chinese Journal of Evidence Based Pediatrics

基　　金：中央高水平医院临床科研业务费资助(北京大学第一医院高质量临床研究专项:2023HQ02)。

摘　　要：背景利妥昔单抗(RTX)是治疗儿童原发肾病综合征频复发或激素依赖的一种激素助减剂,然而该药用于儿童激素耐药肾病综合征(SRNS)的疗效及安全性尚不明确。目的评估RTX用于儿童SRNS的疗效及安全性。设计系统评价和Meta分析。方法检索PubMed、Embase、Cochrane Library、Web of Science、中华医学期刊、中国知网、维普及万方数据库收录的2008至2022年关于RTX治疗儿童SRNS的疗效及安全性的文献,提取相关资料进行分析。主要结局指标SRNS的缓解率及RTX的不良反应。结果10篇文献进入本文分析,总样本量266例。RTX治疗后随访6个月共239例,总缓解例数(包括完全缓解和部分缓解)115例(48.12%)。微小病变(MCD)总体缓解率是局灶节段肾小球硬化(FSGS)的2.3倍(RR=2.30,95%CI:1.46~3.63),差异有统计学意义。其中随访6个月MCD的总体缓解率是FSGS的1.96倍(RR=1.96,95%CI:1.09~3.53),差异有统计学意义;应用RTX时正常尿蛋白组完全缓解率是蛋白尿组的2.83倍(RR=2.83,95%CI:1.57~5.09),差异有统计学意义。多药耐药(MDR)总的缓解率(完全缓解和部分缓解)为36%(95%CI:28%~44%),其中联合用药组和单一用药组分别为31%(95%CI:26%~37%)和44%(95%CI:25%~51%)、部分缓解率为33%(95%CI:22%~44%),初治激素耐药组缓解率为28%(95%CI:17%~41%)、迟发激素耐药组缓解率为50%(95%CI:31%~70%)。激素-钙调磷酸抑制剂(CNI)耐药缓解率为30%(95%CI:16%~46%)。输液反应是RTX最常见的不良反应,其次是感染。结论部分SRNS患儿应用RTX可以获得缓解,且耐受性良好。应用RTX前的病理类型、尿蛋白程度及SRNS临床特征可影响RTX的疗效。Background As a steroid-sparing agent,Rituximab(RTX)has been recommended to treat children with frequently relapsing or steroid-dependent nephrotic syndrome.However,the efficacy and safety of RTX in children with steroid-resistant nephrotic syndrome(SRNS)are not clear.Objective To evaluate the efficacy and safety of RTX in children with SRNS.Design Systematic review and meta-analysis.Methods To retrieve the literature of the efficacy and safety of RTX in children with SRNS,published during the period of 2008 to 2022 in public databases including PubMed,Embase,Cochrane Library,Web of Science,Chinese Medical Journal,China National Knowledge Internet,China Science and Technology Journal Database and wan Fang Data,and to extract the relevant data for analysis.Main outcome measures Remission rates of SRNS and adverse reactions of RTX.Results Ten qualified literatures with a total sample size of 266 cases were included in the study.Among 239 patients who had 6-month follow-up after RTX treatment,115 cases(48.12%)of remission including complete and partial remission.were observed.The overall remission rate of minimal change disease(MCD)was 2.30 times higher than that of focal segmental glomerulosclerosis(FSGS)(RR=2.30,95%CI:1.46-3.63),and the difference was statistically significant.The overall remission rate of MCD at 6-month follow-up was 1.96 times higher than that of FSGS(RR=1.96,95%CI:1.09-3.53),and the difference was statistically significant.The complete remission rate of the normal proteinuria group was 2.83 times higher than that of the proteinuria group(RR=2.83,95%CI:1.57-5.09),and the difference was statistically significant.The overall remission rate(complete remission and partial remission)of multi-drug-resistant(MDR)group was 36%(95%CI:28%-44%),including 31%(95%CI:26%-37%)and 44%(95%CI:25%-65%)in the combination and monotherapy subgroups,respectively.The complete remission rate was 36%(95%CI:23%-50%),including 29%(95%CI:22%-37%)and 62%(95%CI:52%-72%)in the combination and monotherapy subgroups,respectively

关 键 词：利妥昔单抗 激素耐药肾病综合征 儿童 

分 类 号：R726.9[医药卫生—儿科]