过敏性哮喘患儿使用奥马珠单抗的有效性和安全性评估:中国真实世界中的回顾性多中心研究  

Efficacy and safety of Omalizumab for the treatment of pediatric allergic asthma:a retrospective multicenter real-world study in China

作　　者：向莉[1] 徐保平[2] 皇惠杰 魏勉 陈德晖[3] 翟莺莺[3] 张影菊[4] 梁丹[4] 何春卉 侯伟[6] 张洋[6] 陈志敏[7] 刘金玲[7] 刘长山[8] 王雪艳[8] 华山 张宁 李明[10] 张泉[10] 叶乐平 丁玮 周薇[12] 刘玲[12] 王灵[13] 全莹禹[13] 陈艳萍[14] 孟燕妮[14] 葛秋生[15] 张琪 陈杰[16] 王桂兰[17] 黄东明[17] 殷勇[18] 唐铭钰[18] 申昆玲[2] Xiang Li;Xu Baoping;Huang Huijie;Wei Mian;Chen Dehui;Zhai Yingying;Zhang Yingju;Liang Dan;HeChunhui;Hou Wei;Zhang Yang;Chen Zhimin;Liu Jingling;Liu Changshan;Wang Xueyan;Hua Shan;ZhangNing;Li Ming;Zhang Quan;Ye Leping;Ding Wei;Zhou Wei;Liu Ling;Wang Ling;Quan Yingyu;ChenYanping;Meng Yanni;Ge Qiusheng;Zhang Qi;Chen Jie;Wang Guilan;Huang Dongming;Yin Yong;TangMingyu;Shen Kunling(China National Clinical Research Center for Respiratory Diseases,Department of Allergy,Beijing Children′s Hospital,Capital Medical University,National Center for Children′s Health,Beijing 100045,China;China National Clinical Research Center for Respiratory Diseases,Department of Respiratory,Beijing Children′s Hospital,Capital Medical University,National Center for Children′s Health,Beijing 100045,China;Department of Pediatrics,the First Affiliated Hospital of Guangzhou Medical University,Guangzhou 510120,China;Department of Pediatrics,Chengdu First People′s Hospital,Chengdu 610095,China;Department of Respiratory,Guangzhou Women and Children′s Medical Center,Guangzhou 510623,China;Department of Respiratory,the Second Affiliated Hospital of Xi′an Jiaotong University,Xi′an 710004,China;Department of Pulmonology,Children′s Hospital,Zhejiang University School of Medicine,National Clinical Research Center for Child Health,Hangzhou 310052,China;Department of Pediatrics,the Second Hospital of Tianjin Medical University,Tianjin Medical University Children′s Respiratory and Asthma Research Center,Tianjin 300211,China;Department of Respiratory,Anhui Provincial Children′s Hospital,Hefei 230022,China;Department of Respiratory,Kunming Children′s Hospital,Kunming 650103,China;Department of Pediatrics,Peking University First Hospital,Beijing 100034,China;Department of Pediatrics,Peking University Third Hospital,Beijing 100191,China;Department of Pediatrics,Hainan General Hospital,Haikou 570102,China;Department of Respiratory,Hunan Children′s Hospital,Changsha 410001,China;Department of Pediatrics,Peking University Shenzhen Hospita

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过敏反应科,国家呼吸系统疾病临床医学研究中心,北京100045 [2]国家儿童医学中心,首都医科大学附属北京儿童医院呼吸科,国家呼吸系统疾病临床医学研究中心,北京100045 [3]广州医科大学附属第一医院儿科,广州510120 [4]成都市第一人民医院儿科,成都610095 [5]广州市妇女儿童医疗中心呼吸科,广州510623 [6]西安交通大学第二附属医院儿科,西安710004 [7]国家儿童健康与疾病临床医学研究中心,浙江大学医学院附属儿童医院呼吸内科,杭州310052 [8]天津医科大学第二医院儿科,天津医科大学儿童呼吸及哮喘研究中心,天津300211 [9]安徽省儿童医院呼吸内科,合肥230022 [10]昆明市儿童医院呼吸内科,昆明650103 [11]北京大学第一医院儿科,北京100034 [12]北京大学第三医院儿科,北京100191 [13]海南省人民医院小儿内科,海口570102 [14]湖南省儿童医院呼吸内科,长沙410001 [15]北京大学深圳医院儿科,深圳518036 [16]中日友好医院儿科,北京100029 [17]南方医科大学附属中山市博爱医院小儿呼吸内科,中山528400 [18]上海交通大学医学院附属上海儿童医学中心呼吸科,上海200127

出　　处：《中华实用儿科临床杂志》2023年第1期64-71,共8页Chinese Journal of Applied Clinical Pediatrics

基　　金：国家呼吸系统疾病临床医学研究中心"呼吸专项"(HXZX-20210203、HXZX-20210204、HXZX-202107)北京市医院管理中心儿科学科协同发展中心专项经费资助项目(XTCX201818)

摘　　要：目的在中国真实世界中评估过敏性哮喘患儿使用奥马珠单抗的有效性与安全性。方法回顾性分析2018年7月6日至2020年9月30日在中国17家医院接受奥马珠单抗治疗的6~11岁过敏性哮喘患儿的临床资料。采集数据包括患儿接受治疗前的人口学特征、过敏史、家族史、总免疫球蛋白E(IgE)与特异性IgE、皮肤点刺试验、呼出气一氧化氮(FeNO)水平、嗜酸性粒细胞(EOS)计数、合并症;描述性分析奥马珠单抗治疗模式,包括首次剂量、注射间隔频率和疗程与说明书推荐模式的差异;分析奥马珠单抗治疗后疗效整体评估(GETE),比较治疗前后中重度哮喘急性发作率的差异、吸入糖皮质激素(ICS)剂量的变化、肺功能变化,基线与奥马珠单抗治疗后第4、8、12、16、24、52周时儿童哮喘控制测试(C-ACT)和儿童哮喘生活质量问卷(PAQLQ)的变化、合并症改善;安全性评估数据包括不良事件(AE)与严重不良事件(SAE)。对"中重度哮喘年化发作率"以及"ICS减量"指标采用t检验进行差异分析,显著水平为0.05。其他指标均为描述性分析。共纳入200例患儿,其中男151例(75.5%),女49例(24.5%);年龄(8.20±1.81)岁。结果200例患儿中位总IgE为513.5(24.4~11600.0)IU/mL,中位治疗时间为112(1~666)d。200例患儿奥马珠单抗首次注射中位剂量为300(150~600)mg,其中114例(57.0%)有说明书推荐剂量。患儿经奥马珠单抗治疗4~6个月有效率为88.5%(117/200)。患儿接受奥马珠单抗治疗4周后C-ACT评分提高[(18.90±3.74)分比(22.70±3.70)分],达到哮喘控制。奥马珠单抗治疗4~6个月后,中重度哮喘急性发作率降低了(2.00±5.68)次/(人·年)(t=4.7025,P<0.001)。中位ICS日剂量降低[0(0~240)μg比160(50~4000)μg],P<0.001。PAQLQ评分提高[(154.90±8.57)分比(122.80±27.15)分]。第1秒用力呼气量占预计值百分比(FEV 1%pred)提高[(92.80±10.50)%比(89.70±18.17)%]。在有合并症(包括过敏性鼻炎、特应性皮炎/湿Objective To assess the clinical effectiveness and safety of Omalizumab for treating pediatric allergic asthma in real world in China.Methods The clinical data of children aged 6 to 11 years with allergic asthma who received Omalizumab treatment in 17 hospitals in China between July 6,2018 and September 30,2020 were retrospectively analyzed.Such information as the demographic characteristics,allergic history,family history,total immunoglobulin E(IgE)levels,specific IgE levels,skin prick test,exhaled nitric oxide(FeNO)levels,eosinophil(EOS)counts,and comorbidities at baseline were collected.Descriptive analysis of the Omalizumab treatment mode was made,and the difference in the first dose,injection frequency and course of treatment between the Omalizumab treatment mode and the mode recommended in the instruction was investigated.Global Evaluation of Treatment Effectiveness(GETE)analysis was made after Omalizumab treatment.The moderate-to-severe asthma exacerbation rate,inhaled corticosteroid(ICS)dose,lung functions were compared before and after Omalizumab treatment.Changes in the Childhood Asthma Control Test(C-ACT)and Pediatric Asthma Quality of Life Questionnaire(PAQLQ)results from baseline to 4,8,12,16,24,and 52 weeks after Omalizumab treatment were studied.The commodity improvement was assessed.The adverse event(AE)and serious adverse event(SAE)were analyzed for the evaluation of Omalizumab treatment safety.The difference in the annual rate of moderate-to-severe asthma exacerbation and ICS reduction was investigated by using t test.The significance level was set to 0.05.Other parameters were all subject to descriptive analysis.A total of 200 allergic asthma patients were enrolled,including 75.5%(n=151)males and 24.5%(n=49)females.The patients aged(8.20±1.81)years.Results The median total IgE level of the 200 patients was 513.5(24.4-11600.0)IU/mL.Their median treatment time with Omalizumab was 112(1-666)days.Their first dose of Omalizumab was 300(150-600)mg.Of the 200 cases,114 cases(57.0%)followed the first

关 键 词：奥马珠单抗 过敏性哮喘 儿童 有效性 安全性 症状控制 急性加重 激素减量 合并过敏性疾病 

分 类 号：R725.6[医药卫生—儿科]