艾加莫德α治疗全身型重症肌无力的快速卫生技术评估

Efgartigimod alfa in the treatment of generalized myasthenia gravis:a rapid health technology assessment

目的对艾加莫德α治疗全身型重症肌无力的有效性、安全性和经济性进行快速卫生技术评估,为临床合理用药提供循证依据。方法计算机检索PubMed、Embase、Web of Science、Cochrane Library、CNKI、VIP、WanFang Data、SinoMed数据库及卫生技术评估机构官方网站,搜集艾加莫德α治疗全身型重症肌无力的卫生技术评估报告、系统评价/Meta分析和药物经济学研究,检索时限为建库至2024年6月21日。由2名研究者筛选文献、提取数据、评价文献质量,将研究结果汇总并进行定性描述分析。结果共纳入文献6篇,包括5篇系统评价/Meta分析和1篇药物经济学研究。有效性方面,与安慰剂相比,艾加莫德α可显著降低全身型重症肌无力患者的重症肌无力日常生活能力评分、重症肌无力定量评分和15项重症肌无力生活质量评分,差异有统计学意义(P<0.05),但与巴托利单抗、洛利昔珠单抗、依库珠单抗等其他生物制剂相比,各研究得出的结论不一致。安全性方面,艾加莫德α组与安慰剂组成年全身型重症肌无力患者的不良事件发生率差异无统计学意义(P>0.05),但与其他生物制剂相比,各研究得出的结论不一致。经济学方面,与传统疗法相比,艾加莫德α不具有成本-效果优势。结论艾加莫德α治疗全身型重症肌无力的疗效优于安慰剂,但与其他生物制剂相比无确切结论。在安全性方面,艾加莫德α在成年全身型重症肌无力患者中不良事件发生率与安慰剂相当,与其他生物制剂相比无确切结论。根据美国药物经济学数据,与传统疗法相比,艾加莫德α不具有经济学优势。

Objective To evaluate the efficacy,safety and economy of efgartigimod alfa in the treatment of generalized myasthenia gravis by rapid health technology assessment,and to provide evidence-based evidence for clinical rational drug use.Methods PubMed,Embase,Web of Science,Cochrane Library,CNKI,VIP,WanFang Data,SinoMed database and relevant databases of health technology assessment institutions were electronically searched to collect health technology assessment reports,systematic reviews/Meta-analysis and pharmacoeconomic literatures of efgartigimod alfa for the treatment of generalized myasthenia gravis from the inception to June 21,2024.Two researchers screened the literature,extracted data,and evaluated the quality of the literature,summarised the finding and performed qualitative descriptive analysis.Results A total of 6 literature were included,involving 5 systematic reviews/Meta-analysis and one pharmacoeconomic study.In terms of efficacy,efgartigimod alfa demonstrated a significant reduction in MG-ADL,QMGs,and MG-QOLs 15R compared to placebo among generalized myasthenia gravis patients,these differences were statistically significant(P<0.05).However,findings from different studies regarding comparisons with other biologics like batoclimab,ronzanolixizumab,and eculizumab yielded inconsistent conclusions.In terms of safety,the incidence of adverse events in adults generalized myasthenia gravis patients treated with efgartigimod alfa compared with that of the placebo was not statistically significant(P>0.05).However,compared with other biological agents,the conclusions drawn from each study were inconsistent.In terms of economics,efgartigimod alfa did not exhibit cost-effectiveness advantages over traditional therapies.Conclusion Efgartigimod alpha showed better efficacy than placebo in the treatment of generalized myasthenia gravis,but there was no definitive conclusion compared with other biological agents.In terms of safety,the incidence of ADE in adult generalized myasthenia gravis patients treated with efgartigimod alpha was not statistically different from that of the placebo group,and no definite conclusion could be drawn when compared with other biological agents.According to U.S.drug economic data,efgartigimod alpha does not have economic advantages over traditional therapies.