实效性随机对照试验(Pragmatic Randomized Controlled Trial,PRCT )报告的统一标准(Consolidated Standards of Reporting Trials,CONSORT)扩展版——概述

发布于 2023年5月5日 星期五 21:12:36 浏览:1118
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为了克服“解释性”试验结果外推性不足的局限性,实效性随机对照试验(Pragmatic Randomized Controlled Trial,PRCT)应运而生。PRCT报告的统一标准(Consolidated Standards of Reporting Trials,CONSORT)扩展版旨在为报告此类基于真实世界环境的干预性研究提供指导,是CONSORT声明扩展的重要内容。本文概述PRCT CONSORT扩展版相关内容。

关键词:实效性随机对照试验; 随机对照试验; 报告规范; 临床试验报告的统一标准; CONSORT扩展版

一、PRCT报告规范背景

一项临床试验结果的应用价值同时受到内部真实性和外部真实性的影响。目前大部分试验均在理想条件下进行,因此外推试验结果时可能会受到限制,因此,实效性随机对照试验(Pragmatic randomized controlled trial,PRCT)应运而生。PRCT是指尽可能保证研究对象、干预措施、研究环境等与实际情况相似,且尽可能选用与决策直接相关的指标作为结局的一种研究设计。它可以提高研究的外部真实性,能为循证决策者提供与实践更直接相关的证据,使用前需对PRCT结果的真实性、适用性及干预措施的可行性进行评价。目前主要用于评价干预措施实际效果(effectiveness)的研究。

随着PRCT的关注度越来越高,2008年底PRCT的CONSORT扩展版声明发表。PRCT的CONSORT扩展版声明在CONSORT 2001声明的基础上,结合PRCT特点,对第2、3、4、6、7、11、13和21等八项条目进行了补充修订,是目前公认的PRCT报告规范。

二、实效性随机对照试验CONSORT扩展版条目清单

1 实效性临床试验CONSORT扩展版的条目清单中英文对照

内容条目 序号标准清单内容PRCT扩展版内容
Title and abstract
标题与摘要
1How participants were allocated to interventions (e.g., "random allocation" "randomised" or "randomly assigned")
如何把患者分配到不同的干预组(如“随机分配”“随机化”)
 
Introduction
引言
Background
背景
2Scientific background and explanation of rationale
论述研究背景和解释相关原理
Describe the health or health service problem that the intervention is intended to address and other interventions that may commonly be aimed at this problem
说明某项干预措施所针对的健康或健康服务问题,以及针对这一问题的其他干预措施
Methods
方法
Participants
研究对象
3Eligibility criteria for participants; settings and locations where the data were collected
报告纳入标准、收集数据的环境和地点
Eligibility criteria should be explicitly framed to show the degree to which they include typical participants and/or, where applicable, typical providers (eg. nurses), institutions (eg, hospitals), communities (or localities eg, towns) and settings of care (eg, different healthcare financing systems)
清楚描述研究对象的纳入标准,如果可能的话,还应描述干预实施者(如护士)、研究机构(如医院)、社区或居住区(如城镇)和医疗环境(如不同的医疗保险体系)
Interventions
干预
4Precise details of the interventions intended for each group and how and when they were actually administered
准确描述每组的干预,以及实施干预的过程和时间
Describe extra resources added to (or resources removed from) usual settings inorder to implement intervention. Indicate if efforts were made to standardise the intervention or if the intervention and its delivery were allowed to vary between participants, practitioners,or study sites. Describe the comparator in similar detail to the intervention
描述实施干预的过程中所存在的比实际环境多消耗或节省的资源。指出是否将干预措施标准化,是否允许不同的研究对象、实施者或研究地点采取不同的干预措施和途径。用同样的方式描述对照组。
Objectives
目的
5Specific objectives and hypotheses
明确说明目标和假设
 
Outcomes
结局
6Clearly defined primary and secondary outcome measures and, when applicable, any methods used to enhance the quality of measurements (eg, multiple observations, training of assessors)
清楚定义主要结局和次要结局,如果可以的话,还应报告提高结局测量质量的方法(如多次测量,测量者培训)
Explain why the chosen outcomes and, when relevant, the length of follow-up are considered important to those who will use the results of the trial
解释为什么所选结局及随访时间(如果相关的话)对于使用试验结果的人来说是重要的
Sample size
样本量
7How sample size was determined; explanation of any interim analyses and stopping rules when applicable
报告如何确定样本量,可以的话还应该报告中期分析和终止试验的规则
If calculated using the smallest difference considered important by the target decision maker audience (the minimally important difference) then report where this difference was obtained
如果计算样本量时使用的是基于决策者所认为的最小差异(最小重要差异),那么需报告该差异如何获得的
Randomisation-sequence generation
随机分配序列的产生
8Method used to generate the random allocation sequence, including details of any restriction (eg, blocking, stratification)
报告产生随机分配序列的方法,包括任何限制性随机化的细节(如区组、分层)
 
Randomisation-allocation concealment
分组隐匿
9Method used to implement the random allocation sequence (eg, numbered containers or central telephone), clarifying whether the sequence was concealed until interventions were assigned
报告实施随机的方法(如编码容器或中心电话),明确在实施干预之前分配是否隐藏
 
Randomisation-implementation
随机化实施
10Who generated the allocation sequence, who enrolled participants, and who assigned participants to their groups
说明由谁生成随机分配序列,谁招募研究对象,谁将研究对象进行分组
 
Blinding
盲法
11Whether or not participants, those administering the interventions, and those assessing the outcomes were blinded to group assignment. If done, how the success of blinding was evaluated
报告研究对象、干预实施者、结局评估者是否不知道分组情况
If blinding was not done, or was not possible, explain why
如果没有实施盲法,或者无法实施盲法,解释其原因
Statistical methods
统计学方法
12Statistical methods used to compare groups for primary outcomes; methods for additional analyses, such as subgroup analyses and adjusted analyses
说明比较主要结局指标的统计方法;其他分析方法,如亚组分析和调整分析
 
Results
结果
Participant flow
研究对象纳入流程
13Flow of participants through each stage (a diagramis strongly recommended)—specifically, for eachgroup, report the numbers of participants randomly assigned, receiving intended treatment, completing the study protocol, and analysed for the primary outcome; describe deviations from planned study protocol, together with reasons
报告每一阶段参与试验的研究对象人数(建议使用流程图)—即每个组的随机分配的人数、接受治疗的人数、完成治疗方案的人数、用于主要结局分析的人数等。对于违背研究计划之处,还要对其原因加以解释
The number of participants or units approached to take part in the trial, the number which were eligible, and reasons for non-participation should be reported
报告参加试验的研究对象或单位的数目、合格的数目以及未参与试验的原因
Recruitment
招募
14Dates defining the periods of recruitment and follow-up
说明招募和随访的日期
 
Baseline data
基线数据
15Baseline demographic and clinical characteristics of each group
描述每组的人口统计学基线资料和临床特征
 
Number analyzed
分析例数
16Number of participants (denominator) in each group included in each analysis and whether analysis was by "intention-to-treat"; state the results in absolute numbers when feasible (eg, 10/20, not 50%)
每项分析中都需要报告每组分析的研究对象人数(分母),注明是否采用了意向性分析;如果可能,应该采用绝对数报告结果(例如10/20,而非50%)
 
Outcomes and estimation
结局和效应估计
17For each primary and secondary outcome, a asummary of results for each group and the estimated effect size and its precision (eg, 95% CI)
对于每一项主要结局和次要结局,需报告每组的结果、效应值及其精确度(如95%置信区间)
 
Ancillary analyses
其他分析
18Address multiplicity by reporting any other analyses performed, including subgroup analyses and adjusted analyses, indicating which are prespecified and which are exploratory
若处理多因素,应报告其他分析,包括亚组分析和调整分析,并指出哪些是事先设定的,哪些是探索性的
 
Adverse events
不良事件
19All important adverse events or side effects in each intervention group
报告每个组发生的所有重要的不良事件或副作用
 
Discussion
讨论
Interpretation
解释
20Interpretation of the results, taking into account study hypotheses, sources of potential bias or imprecision, and the dangers associated with multiplicity of analyses and outcomes
解释结果时要考虑研究假设、潜在的偏倚或不精确性,以及多重比较和多种结局的问题
 
Generalizability
外推性
21Generalisability (external validity) of the trial findings
解释试验结果的外推性(外部真实性)
Describe key aspects of the setting which determined the trial results. Discuss possible differences in other settings where clinical traditions, health service organization, staffing, or resources may vary from those of the trial
描述影响试验结果的关于研究环境的重要信息。讨论在其他环境下,如临床常规、卫生服务机构、服务人员或资源与试验存在区别时,可能会产生的不同效果
Overall evidence
整体证据
22General interpretation of the results in the context of current evidence
基于当前证据对结果进行概括性解释
 

三、实效性随机对照试验CONSORT扩展版使用注意事项

使用该声明前,研究者应先确认报告的试验是否属于PRCT。一般来说,研究对象的纳入标准越宽泛,干预措施越灵活,对照组接受的干预措施越普遍,结局对决策者越重要,那么试验的外推性就越强,实效性就越大。

近年来有学者提出了实效-解释连续性综合指标(Pragmatic-explanatorycontinuum indicator summary,PRECIS, https://pubmed.ncbi.nlm.nih.gov/19348971/)。PRECIS包括10个维度,即研究对象的入选标准、干预措施的灵活性、干预实施者的专业水平、对照组的干预措施、对照组干预实施者的专业水平、随访频率、主要结局性质、研究对象依从性、实施者对干预方案的遵守程度以及主要结局的分析。通过不同维度之间的连线所围成的轮状图面积大小来评价一项试验实效性和解释性,面积越大,说明该试验实效性越强,面积越小,解释性越强。此外,试验的实效性和解释性与研究目的相关,若研究者希望通过一项试验来评价某种干预的生物学效应,那么这项试验就更倾向于解释性;而若是为了帮助决策者决定是否选用这种干预,那么这项试验就更倾向于实效性。

当试验倾向实效性时,研究者可根据PRCT的CONSORT扩展声明进行报告,根据需要或要求可将PRCT的CONSORT扩展声明清单(checklist)作为投稿材料之一递交,并引用已发表的相关报告规范文献。审稿人也可根据编辑部要求参照PRCT的CONSORT扩展声明标准和作者提供的清单来审稿。决策者可根据该扩展声明对PRCT试验进行报告质量评价。一般CONSORT声明的所有注意事项和局限性同样适用于本指南。

注:本文内容是参考相关文献后对PRCT CONSORT扩展版报告规范原文的概述,仅代表本网站观点。关于实效性随机对照试验CONSORT扩展版的更多内容详见官方网站(http://www.consort-statement.org/downloads/extensions)或Merrick Zwarenstein等发表的论文“Improving the reporting of pragmatic trials: an extension of the CONSORT statement (https://pubmed.ncbi.nlm.nih.gov/19001484/)”。

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