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/湿疹、荨麻疹、过敏性结膜炎、鼻窦炎)评估记录的患儿中,总体改善率为92.8%~100.0%。200例患儿中,58例(29.0%)发生124例次AE,发生率为0(0~15.1)例次/(人·年)。在53例发生AE且报告了严重程度的患儿中,83.0%(44例)发生轻度AE,17.0%(9例)发生中度AE,无重度AE发生;SAE的发生率为0(0~1.9)例次/(人·年)。与药物相关常见AE为腹痛(2例患儿,1.0%)和发热(2例患儿,1.0%),未出现因AE而停药。结论奥马珠单抗治疗中重度过敏性哮喘患儿有效率高,可降低中重度哮喘急性发作、减少ICS剂量、提高哮喘控制水平、改善肺功能及生活质量,且其安全性良好。
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 Omalizumab dosage recommended in the instruction.After 4-6 months of Omalizumab treatment,88.5%of the patients enrolled(n=117)responded to Omalizumab.After 4 weeks of treatment with Omalizumab,asthma was well-controlled,with an increased C-ACT score[from(22.70±3.70)points to(18.90±3.74)points at baseline].Four-six months after Omalizumab administration,the annual rate of moderate-to-severe asthma exacerbation had a reduction of(2.00±5.68)per patient year(t=4.7025,P<0.001),the median ICS daily dose was lowered[0(0-240)μg vs.160(50-4000)μg at baseline](P<0.001),the PAQLQ score was improved[(154.90±8.57)points vs.(122.80±27.15)points at baseline],and the forced expiratory volume in one second%predicted(FEV 1%pred)was increased[(92.80±10.50)%vs.(89.70±18.17)%at baseline].In patients with available evaluations for comorbidities,including allergic rhinitis,atopic dermatitis or eczema,urticaria,allergic conjunctivitis and sinusitis,92.8%-100.0%showed improved symptoms.A total of 124 AE were reported in 58(29.0%)of the 200 patients,and the annual incidence was 0(0-15.1)per patient year.In 53 patients who suffered AE,44 patients(83.0%)and 9 patients(17.0%)reported mild and moderate AE,respectively.No severe AE were observed in patients.The annual incidence of SAE was 0(0-1.9)per patient year.Most common drug-related AE were abdominal pain(2 patients,1.0%)and fever(2 patients,1.0%).No patient withdrew Omalizumab due to AE.Conclusions Omalizumab shows good effectiveness and safety for the treatment of asthma in children.It can reduce the moderate-to-severe asthma exacerbation rate,reduce the ICS dose,improve asthma control levels,and improve lung functions and quality of life of patients.
作者
向莉
徐保平
皇惠杰
魏勉
陈德晖
翟莺莺
张影菊
梁丹
何春卉
侯伟
张洋
陈志敏
刘金玲
刘长山
王雪艳
华山
张宁
李明
张泉
叶乐平
丁玮
周薇
刘玲
王灵
全莹禹
陈艳萍
孟燕妮
葛秋生
张琪
陈杰
王桂兰
黄东明
殷勇
唐铭钰
申昆玲
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 Hospital,Shenzhen 518036,China;Department of Pediatrics,China-Japan Friendship Hospital,Beijing 100029,China;Department of Pediatric Respiratory,Zhongshan Boai Hospital of Southern Medical University,Zhongshan 528400,China;Department of Respiratory Medicine,Shanghai Children′s Medical Center Affiliated to Shanghai Jiaotong University School of Medicine,Shanghai 200127,China)
出处
《中华实用儿科临床杂志》
CAS
CSCD
北大核心
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)
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
Omalizumab
Allergic asthma
Child
Effectiveness
Safety
Symptom control
Exacerbation
Corticosteroid dosage reduction
Allergic comorbidities
