Handling missing data in RCTs; a review of the top medical journals
Handling missing data in RCTs; a review of the top medical journals
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DOI：
10.1186/1471-2288-14-118
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发表时间：
2014-11-19
影响因子：
4
通讯作者：
Hsu, Chiu-Hsieh
中科院分区：
文献类型：
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作者：
Bell, Melanie L.;Fiero, Mallorie;Hsu, Chiu-Hsieh
背景：在随机对照试验中，缺少结局数据对治疗效果估计的有效性构成威胁。我们的目的是评估顶级医学期刊上随机对照试验（rct）缺失数据的程度、处理和敏感性分析以及意向治疗（ITT）分析，并将我们的研究结果与之前关于随机对照试验中缺失数据和ITT的综述进行比较。方法：回顾2013年7月至12月间发表在BMJ、JAMA、Lancet和New England Journal of Medicine上的随机对照试验，排除聚类随机试验和以生存为主要结局的试验。结果：在77篇确定的符合条件的文章中，73篇（95%）报告了一些缺失的结局数据。缺失结果的参与者中位数百分比为9%（范围0 - 70%）。在初级分析中，最常用的缺失数据处理方法是完整案例分析（33,45%），其次是简单方法代入20例（27%），基于模型方法代入15例（19%），多重方法代入6例（8%）。27个（35%）缺少数据的试验报告了敏感性分析。然而，大多数人并没有改变原始分析中缺失数据的假设。在52项（85%）试验中发现了ITT或修改ITT的报告，其中21项（40%）试验包括所有随机受试者。与2001年报告的一项试验审查的比较表明，数据丢失率和方法相似，但ITT一词的使用有所增加，敏感性分析报告也有所增加。结论：缺少结果数据仍然是随机对照试验中的一个常见问题。ITT方法的定义在不同的试验中仍然不一致。与缺失数据相关的统计方法研究与这些方法在应用环境中的使用之间存在明显的巨大差距，包括顶级医学期刊上的随机对照试验。
Background: Missing outcome data is a threat to the validity of treatment effect estimates in randomized controlled trials. We aimed to evaluate the extent, handling, and sensitivity analysis of missing data and intention-to-treat (ITT) analysis of randomized controlled trials (RCTs) in top tier medical journals, and compare our findings with previous reviews related to missing data and ITT in RCTs.Methods: Review of RCTs published between July and December 2013 in the BMJ, JAMA, Lancet, and New England Journal of Medicine, excluding cluster randomized trials and trials whose primary outcome was survival.Results: Of the 77 identified eligible articles, 73 (95%) reported some missing outcome data. The median percentage of participants with a missing outcome was 9% (range 0 - 70%). The most commonly used method to handle missing data in the primary analysis was complete case analysis (33, 45%), while 20 (27%) performed simple imputation, 15 (19%) used model based methods, and 6 (8%) used multiple imputation. 27 (35%) trials with missing data reported a sensitivity analysis. However, most did not alter the assumptions of missing data from the primary analysis. Reports of ITT or modified ITT were found in 52 (85%) trials, with 21 (40%) of them including all randomized participants. A comparison to a review of trials reported in 2001 showed that missing data rates and approaches are similar, but the use of the term ITT has increased, as has the report of sensitivity analysis.Conclusions: Missing outcome data continues to be a common problem in RCTs. Definitions of the ITT approach remain inconsistent across trials. A large gap is apparent between statistical methods research related to missing data and use of these methods in application settings, including RCTs in top medical journals.