大多数时候都搞错了？比较试验者对主要结局的选择与患者和卫生专业人员的需求。
Getting it wrong most of the time? Comparing trialists' choice of primary outcome with what patients and health professionals want.
机构信息
Health Services Research Unit, University of Aberdeen, Aberdeen, UK.
Janssen-Cilag Farmacêutica Ltda, São Paulo, Brazil.
出版信息
Trials. 2022 Jun 27;23(1):537. doi: 10.1186/s13063-022-06348-z.
BACKGROUND
Randomised trials support improved decision-making through the data they collect. One important piece of data is the primary outcome - so called because it is what the investigators decide is the most important. Secondary outcomes provide additional information to support decision-making. We were interested in knowing how important patients and healthcare professionals consider the outcomes (especially the primary outcome) measured in a selection of published trials.
METHODS
The work had three stages: (1) We identified a body of late-stage trials in two clinical areas, breast cancer management and nephrology. (2) We identified the primary and secondary outcomes for these trials. (3) We randomly ordered these outcomes and presented them to patients and healthcare professionals (with experience of the clinical area), and we asked them to rank the importance of the outcomes. They were not told which outcomes trial authors considered primary and secondary.
RESULTS
In our sample of 44 trials with 46 primary outcomes, 29 patients, one patient representative and 12 healthcare professionals together ranked the primary outcome as the most important outcome 13/46 times or 28%. Breast cancer patients and healthcare professionals considered the primary outcome to be the most important outcome for 8/21 primary outcomes chosen by trialists. For nephrology, the equivalent figure was 5/25. The primary outcome appeared in a respondent's top 5 ranked outcomes 151/178 (85%) times for breast cancer and 225/259 (87%) times for nephrology even if the primary was not considered the most important outcome.
CONCLUSIONS
The primary outcome in a trial is the most important piece of data collected. It is used to determine how many participants are required, and it is the main piece of information used to judge whether the intervention is effective or not. In our study, patients and healthcare professionals agreed with the choice of the primary outcome made by trial teams doing late-stage trials in breast cancer management and nephrology 28% of the time.
背景
随机试验通过收集的数据支持改善决策。一个重要的数据是主要结局——之所以这样称呼，是因为它是研究者认为最重要的结果。次要结局提供了额外的信息以支持决策。我们想知道患者和医疗保健专业人员如何看待在一系列已发表的试验中测量的结果（特别是主要结局）。
方法
这项工作分三个阶段进行：（1）我们在两个临床领域（乳腺癌管理和肾脏病学）中确定了一组晚期试验。（2）我们确定了这些试验的主要和次要结局。（3）我们随机排列这些结局，并将其呈现给患者和医疗保健专业人员（具有临床领域经验），并要求他们对结局的重要性进行排名。他们没有被告知哪些结局是试验作者认为的主要和次要结局。
结果
在我们的 44 项试验和 46 个主要结局的样本中，29 名患者、1 名患者代表和 12 名医疗保健专业人员一起将主要结局评为最重要的结局 13/46 次或 28%。乳腺癌患者和医疗保健专业人员认为试验者选择的 21 个主要结局中的 8/21 个主要结局是最重要的结局。对于肾脏病学，这一数字为 5/25。对于乳腺癌，主要结局出现在受访者排名前 5 的结局中 151/178（85%）次，对于肾脏病学，主要结局出现在受访者排名前 5 的结局中 225/259（87%）次，即使主要结局不是最重要的结局。
结论
试验中的主要结局是收集的最重要的数据。它用于确定需要多少参与者，并且是用于判断干预是否有效的主要信息。在我们的研究中，在乳腺癌管理和肾脏病学的晚期试验中，患者和医疗保健专业人员同意试验团队选择的主要结局的情况占 28%。
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