心血管治疗类医疗器械非劣效性试验特点及界值设置的系统评价  

Characteristics and threshold setting of non-inferiority trials for cardiovascular therapeutic medical devices:a systematic review

作　　者：冯堃 郭琼 钟文涛 刘悦 刘宇琪 冷柔菲 许译涵 顾先林 龙囿霖 杜亮[1,2,3,5] 李正赤[5,6] 黄进[2] FENG Kun;GUO Qiong;ZHONG Wentao;LIU Yue;LIU Yuqi;LENG Roufei;XU Yihan;GU Xianlin;LONG Youlin;DU Liang;LI Zhengchi;HUANG Jin(Chinese Evidence-Based Medicine Center,West China Hospital of Sichuan University,Chengdu 610041,P.R.China;Medical Device Regulatory Research and Evaluation Center,West China Hospital of Sichuan University,Chengdu 610041,P.R.China;West China Publishers,West China Hospital of Sichuan University,Chengdu 610041,P.R.China;West China School of Public Health,Sichuan University,Chengdu 610041,P.R.China;Center for Education of Medical Humanities,West China Hospital of Sichuan University,Chengdu 610041,P.R.China;Institute of Hospital Management,West China Hospital of Sichuan University,Chengdu 610041,P.R.China)

机构地区：[1]四川大学华西医院中国循证医学中心,成都610041 [2]四川大学华西医院医疗器械监管研究与评价中心,成都610041 [3]四川大学华西医院华西期刊社,成都610041 [4]四川大学华西公共卫生学院,成都610041 [5]四川大学华西医院医学人文教育中心,成都610041 [6]四川大学华西医院医院管理研究所,成都610041

出　　处：《中国循证医学杂志》2024年第3期288-294,共7页Chinese Journal of Evidence-based Medicine

基　　金：国家自然科学基金项目(编号:72074161)。

摘　　要：目的系统评价心血管领域以医疗器械为干预的非劣效性试验,调查其试验特征和界值设置情况。方法计算机检索PubMed、Embase、CENTRAL数据库,搜集心血管领域医疗器械类的非劣效性试验,检索时限均为建库至2023年7月26日。由2名研究者独立筛选文献、提取资料后,对纳入研究的基本特征、非劣效性试验特征和界值特征进行报告。研究使用Excel 2020和R 4.2.1软件进行分析。结果共纳入214篇研究,其中167篇(78.0%)干预为冠状动脉支架,试验以双臂设计为主(92.9%),多采用非劣效性绝对界值(96.7%)作为非劣效性的判定依据。150篇(70.1%)研究基于预估的对照组效应值确定非劣效性界值,其中33篇(15.4%)研究未报告预估的对照组效应值来源。非劣效性试验终点结局指标多样且界值设置差异较大,靶病变失败率(2.1%~8.6%)、靶血管失败率(2.5%~19.6%)和主要心血管不良事件发生率(2.1%~10.0%)是研究数量最多的三项定性指标,晚期管腔丢失(0.1~0.4 mm)是研究数量最多的定量指标。所有指标的非劣效性绝对界值转化为相对界值后,范围为1.20~3.67。结论心血管领域医疗器械类的非劣效性试验对相同的结局指标设置的非劣效性界值差异较大,界值设置依据报告不足。Objective To systematically review the non-inferiority trials in the cardiovascular domain that utilize medical devices as interventions,and investigate its characteristics and threshold settings.Methods The PubMed,Embase,and CENTRAL databases were electronically searched to collect non-inferiority trials in the cardiovascular field involving medical devices from inception to July 26,2023.Two reviewers independently screened literature and extracted data.The reported information included basic characteristics,features of non-inferiority trials,and threshold-setting features of the included studies.Data analysis was performed using Excel 2020 and R 4.2.1 software.Results A total of 214 studies were included,with 167 studies(78.0%)focusing on interventions related to coronary artery stents.The trials predominantly utilized a two-arm design(92.9%),with a prevalent use of non-inferiority absolute thresholds(96.7%)as the criteria for non-inferiority determination.In 150 studies(70.1%),non-inferiority thresholds were established based on estimated control group effect values,while 33 studies(15.4%)did not report the source of these values.The noninferiority trial endpoint outcomes exhibited diversity,and there were substantial differences in threshold settings.The three most studied qualitative indicators were target lesion failure rates(2.1%-8.6%),target vessel failure rates(2.5%-19.6%),and major adverse cardiovascular events rates(2.1%-10.0%).Late lumen loss(0.1-0.4 mm)emerged as the most frequently studied quantitative indicator.After converting absolute non-inferiority thresholds for all indicators into relative thresholds,the range was 1.20-3.67.Conclusion Significant variations in non-inferiority threshold settings are observed for identical endpoint outcomes across included studies,highlighting a lack of reporting on the rationale behind threshold settings.

关 键 词：治疗类医疗器械 心血管领域医疗器械 非劣效性试验 非劣效性界值 系统评价 

分 类 号：R197.39[医药卫生—卫生事业管理]