Evaluating how clear the questions being investigated in randomised trials are: systematic review of estimands.
Evaluating how clear the questions being investigated in randomised trials are: systematic review of estimands.
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评估随机试验中正在研究的问题的清晰度：对代谢物的系统评价。
DOI：
10.1136/bmj-2022-070146
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发表时间：
2022-08-23
影响因子：
105.7
通讯作者：
Cornelius, Victoria
中科院分区：
文献类型：
--
作者：
Cro, Suzie;Kahan, Brennan C.;Rehal, Sunita;Ster, Anca Chis;Carpenter, James R.;White, Ian R.;Cornelius, Victoria
评估关于干预措施（被估量）的精确研究问题被陈述或可以从报告的方法中确定的频率，并确定在2-4期随机试验中正在研究的问题类型。对2020年在六种领先的综合医学期刊上进行的随机试验中研究的研究问题的清晰度进行了系统综述。PubMed检索，2021年2月。2-4期随机试验，对医疗条件或干预措施没有限制。排除了随机分组、交叉、非劣效性和等效性试验。说明了关于干预措施的确切主要问题（即主要被估量），或者可以使用统计知识从报告的方法中明确确定主要被估量的试验数量。用于处理影响患者结局解释或存在的随机化后事件的策略，例如干预停止或使用额外药物治疗（称为并发事件），以及正在研究的相应问题类型。确定了255项合格的随机试验。没有试验明确说明被估量的所有属性。在255项试验中，有117项（46%）的主要被估量可以从报告的方法中确定。255项试验中有242项（95%）报告了并发事件;但仅在255项试验中的125项（49%）中确定了这些事件的处理。大多数提供这一信息的试验认为并发事件的发生与治疗效果的计算无关，并评估了干预的效果（96/125，77%）-也就是说，他们使用了治疗策略。在99项因不良事件导致治疗不依从的试验中，有4项（4%）估计了假设环境中的治疗效果（即，尽管发生不良事件，但参与者仍继续治疗的效果），在24项患者死亡的试验中，有19项（79%）估计了假设环境中的治疗效果（即，参与者没有死亡的效果）。在大多数试验中正在调查的确切的研究问题是不清楚的，主要是因为缺乏明确的方法来处理并发事件。在试验报告中必须明确报告被估量，以便所有利益相关者，包括临床医生、患者和政策制定者，能够就医疗干预措施做出充分知情的决定。繁荣CRD 42021238053。
To evaluate how often the precise research question being addressed about an intervention (the estimand) is stated or can be determined from reported methods, and to identify what types of questions are being investigated in phase 2-4 randomised trials. Systematic review of the clarity of research questions being investigated in randomised trials in 2020 in six leading general medical journals. PubMed search in February 2021. Phase 2-4 randomised trials, with no restrictions on medical conditions or interventions. Cluster randomised, crossover, non-inferiority, and equivalence trials were excluded. Number of trials that stated the precise primary question being addressed about an intervention (ie, the primary estimand), or for which the primary estimand could be determined unambiguously from the reported methods using statistical knowledge. Strategies used to handle post-randomisation events that affect the interpretation or existence of patient outcomes, such as intervention discontinuations or uses of additional drug treatments (known as intercurrent events), and the corresponding types of questions being investigated. 255 eligible randomised trials were identified. No trials clearly stated all the attributes of the estimand. In 117 (46%) of 255 trials, the primary estimand could be determined from the reported methods. Intercurrent events were reported in 242 (95%) of 255 trials; but the handling of these could only be determined in 125 (49%) of 255 trials. Most trials that provided this information considered the occurrence of intercurrent events as irrelevant in the calculation of the treatment effect and assessed the effect of the intervention regardless (96/125, 77%)—that is, they used a treatment policy strategy. Four (4%) of 99 trials with treatment non-adherence owing to adverse events estimated the treatment effect in a hypothetical setting (ie, the effect as if participants continued treatment despite adverse events), and 19 (79%) of 24 trials where some patients died estimated the treatment effect in a hypothetical setting (ie, the effect as if participants did not die). The precise research question being investigated in most trials is unclear, mainly because of a lack of clarity on the approach to handling intercurrent events. Clear reporting of estimands is necessary in trial reports so that all stakeholders, including clinicians, patients and policy makers, can make fully informed decisions about medical interventions. PROSPERO CRD42021238053.
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影响因子：
158.5
作者：
Kelly, Aaron S.;Auerbach, Pernille;Arslanian, Silva
通讯作者：
Arslanian, Silva
影响因子：
105.7
作者：
Dechartres, Agnes;Trinquart, Ludovic;Ravaud, Philippe
通讯作者：
Ravaud, Philippe
DOI：
10.1136/bmj.m3719
发表时间：
2020-10-14
期刊：
BMJ (Clinical research ed.)
影响因子：
--
作者：
Hagen S;Elders A;Stratton S;Sergenson N;Bugge C;Dean S;Hay-Smith J;Kilonzo M;Dimitrova M;Abdel-Fattah M;Agur W;Booth J;Glazener C;Guerrero K;McDonald A;Norrie J;Williams LR;McClurg D
通讯作者：
McClurg D
影响因子：
2.5
作者：
Kahan BC;Morris TP;White IR;Carpenter J;Cro S
通讯作者：
Cro S
影响因子：
9.3
作者：
Kahan, Brennan C.;Morris, Tim P.;Carpenter, James R.
通讯作者：
Carpenter, James R.