Quality of reporting of noninferiority and equivalence randomized trials
Quality of reporting of noninferiority and equivalence randomized trials
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DOI：
10.1001/jama.295.10.1147
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发表时间：
2006-03-08
影响因子：
120.7
通讯作者：
Ravaud, P
中科院分区：
文献类型：
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作者：
Le Henanff, A;Giraudeau, B;Ravaud, P
背景非劣性和等效性试验旨在表明实验治疗在临床上并不比对照有效治疗差(非劣)或临床类似(相当)。目的评价非劣势和等效性随机对照试验报告的方法学质量。设计检索MEDLINE和Cochrane中央对照试验数据库，检索2003年1月1日至2004年12月31日发表的非劣效性和等效性随机对照试验报告。采用标准化表格提取的主要结果测量数据包括非劣效性或等效性选择的评估、样本量计算、被分析的患者组、统计检验和报告结果的方法以及结论。大多数报告(156例[96.3%])描述了界定非劣性或等价性的边缘，只有33例(20.4%)的边缘是合理的。近四分之一的报告(35份[21.6%])没有描述样本量的计算，另外11份报告(6.8%)没有考虑预先规定的非劣性或等价性。不到一半的报告(69[42.6%])同时描述了意向治疗(ITT；所有随机患者都包括在分析中)或改良的ITT(排除从未接受治疗的患者)和按方案(排除未完成治疗的患者)分析，只有大约一半(39[56.5%])描述了这两种类型的结果。136份报告(84.0%)的结果以可信区间显示。只有33篇文章(20.3%)符合非劣效性和等效性试验的报告要求，其中4篇(12.1%)的结论具有误导性。结论非劣效性和等效性试验的报道存在严重缺陷：缺乏非劣效性或等效性试验，只进行ITT(或按方案)分析，结果报告不充分。此外，即使对于满足这些要求的文章，结论有时也具有误导性。
Context Noninferiority and equivalence trials aim to show that the experimental treatment is not clinically worse than (noninferior) or clinically similar to (equivalent) a control active treatment. These study objectives imply particular planning and analysis.Objective To assess the methodologic quality of reports of randomized controlled trials of noninferiority and equivalence.Design We searched MEDLINE and the Cochrane Central Register of Controlled Trials for reports of randomized controlled trials of noninferiority and equivalence hypotheses published between January 1, 2003, and December 31, 2004.Main Outcome Measures Data extracted by use of a standardized form involved assessment of choice of noninferiority or equivalence margins, sample size calculation, sets of patients analyzed, method of statistical testing and reporting results, and conclusions.Results A total of 162 reports were included in the analysis ( 116 reports of noninferiority and 46 of equivalence). The margin defining noninferiority or equivalence was described in most reports (156 [96.3%]), with justification of the margin in only 33 (20.4%). Almost one quarter of the reports (35 [21.6%]) did not describe a sample size calculation, and an additional 11 (6.8%) did not take into account a prespecified noninferiority or equivalence margin. Less than half of the reports (69 [42.6%]) described both an intent-to-treat (ITT; all randomized patients are included in the analysis) or modified ITT (patients who never received treatment are excluded) and per-protocol (patients who did not complete the treatment are excluded) analysis, and only about half of those (39 [56.5%]) described both types of results. Results were displayed with confidence intervals in 136 reports (84.0%). Only 33 articles (20.3%) fulfilled reporting requirements specific to noninferiority and equivalence trials, 4 of them (12.1%) with misleading conclusions.Conclusions Reporting of noninferiority and equivalence trials has important deficiencies: absence of noninferiority or equivalence margin, only an ITT (or a per-protocol) analysis performed, and results not adequately reported. Moreover, even for articles fulfilling these requirements, conclusions are sometimes misleading.