目的 综合评估奥马珠单抗治疗儿童中重度支气管哮喘的有效性、安全性、经济性、适宜性、可及性与创新性，为其在临床的准入决策、合理使用与监测评估提供依据。方法 系统检索PubMed、Embase、the Co-chrane Library、ClinicalTirals。gov、Web of Science、中国知网、万方数据知识服务平台和中国生物医学文献服务系统等数据库，检索时间自数据库建库至2023年11月24日，纳入奥马珠单抗治疗儿童中重度支气管哮喘的卫生技术评估、系统评价/meta分析、药物经济学研究以及其他药品信息数据。结果 最终纳入15篇文献，均为中等质量或低质量研究。评估结果显示，奥马珠单抗可显著降低儿童重度支气管哮喘恶化率、减少激素抢救性治疗剂量;奥马珠单抗组相比常规治疗组的严重不良事件发生风险更低，长期应用的安全性与耐受性良好;各国儿童应用奥马珠单抗的经济性结论不一致，在我国不具有经济性优势。问卷调研与专家咨询结果显示，奥马珠单抗的有效性和创新性高于常规治疗，安全性略高于常规治疗，但经济性、适宜性和可及性均低于常规治疗。结论 奥马珠单抗治疗儿童中重度支气管哮喘具有良好的有效性和安全性，但其在我国不具有经济性优势，适宜性与可及性略弱于常规治疗方案。因此，在儿童中重度支气管哮喘治疗时，需谨慎评估其必要性，权衡利弊后使用。
Comprehensive clinical evaluation of omalizumab in the treatment of moderate-to-severe bronchial asthma in children
Objective To comprehensively evaluate the efficacy,safety,economy,appropriateness,accessibility,and in-novation of omalizumab for the treatment of moderate-to-severe bronchial asthma in children,and to provide a basis for deci-sion-making on access,rational use,and monitoring and evaluation of omalizumab.Methods Literature databases such as PubMed,Embase,the Cochrane Library,ClinicalTirals.gov,Web of Science,CNKI,Wanfang Database,and SinoMed were systematically searched,and the global major health technology assessment(HTA)organizations'official websites,and the search was conducted from the time of database inception to November 24,2023,to include HTA,systematic review/meta-analysis,pharmacoeconomic studies,and other drug information data of omalizumab for the treatment of moderate-to-severe bronchial asthma in children.Results Fifteen articles were finally included,which were moderate low-quality studies.The re-sults of the evaluation suggested that omalizumab significantly reduced the rate of exacerbation of severe asthma and reduced the dose of control medication in children.The safety profile of omalizumab was good overall,with a lower rate of serious ad-verse events compared to the conventional treatment group,and a good safety and tolerability profile for long-term applica-tion.Conclusions on the economics of omalizumab in children were inconsistent across countries,and there was no economic advantage in our country.The results of the questionnaire study and expert consultation suggest that omalizumab is significant-ly more effective and innovative than the conventional treatment group,slightly safer than the conventional treatment group,but less economical,appropriate,and accessible than the conventional treatment group.Conclusions Omalizumab for moder-ate-to-severe bronchial asthma in children has good efficacy and an overall favorable safety profile.However,omalizumab does not have economic advantages in China,and its appropriateness and accessibility are slightly weaker than that of conventional treat-ment options,so its necessity needs to be carefully evaluated and weighed against its advantages and disadvantages when used in the treatment of moderate-to-severe asthma in children.
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