患者报告结局(Patient Reported Outcomes,PRO)临床试验报告的统一标准(Consolidated Standards of Reporting Trials,CONSORT)——概述

发布于 2023年7月13日 星期四 13:01:39 浏览:284
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患者报告结局(patient reported outcomes,PROs)是指直接由患者提供的自身健康状态的主观信息,包括健康相关生活质量、症状、患者满意度和自我效能等。PROs随机对照试验(randomly control test,RCT)已在临床治疗方案的评价与选择、患者依从性评价、卫生政策与相关保险理赔政策的制定等领域广泛应用。目前,CONSORT-PROs扩展版是公认的涉及PROs的RCT的报告规范,本文概述CONSORT-PROs扩展版相关内容。

关键词:患者报告结局; 报告规范; 临床试验报告的统一标准; CONSORT扩展版;CONSORT-PROs

一、患者报告结局临床试验报告规范(CONSORT-PROs扩展版)背景

患者报告结局(patient reported outcomes,PROs)是指直接来自患者、未经医生或其他人员解释或干涉的有关其健康状况和治疗效果评价的结局指标。在临床试验中涉及PROs相关数据的研究逐渐增加。2009年,PROs被美国食品药品安全管理局(U.S.A Food and drug administration, FDA)正式列为临床疗效评价及药物试验的必要报告项目。但目前PROs试验的质量堪忧,试验者尚未形成准确、完整、有效地报告PROs的规范标准。

CONSORT-PROs扩展版是基于CONSORT(临床试验报告的统一标准(Consolidated Standards of Reporting Trials,CONSORT)——概述)而研制的,2012年1月,在英国伦敦会议上各方达成共识并于2013年正式发表,是目前公认的涉及PROs的随机对照试验(randomly control test,RCT)报告规范。最终的 CONSORT-PROs扩展版包括6个部分,共25个条目。其对CONSORT 2010的9个条目进行了补充说明,并确定了以PROs为主要结局指标或关键的次要结局指标的RCT所需报告的 5 个重要条目,分别是:

  • 在摘要中列出作为主要或次要结局的PRO
  • 陈述PRO相关假设及相关维度(如使用多维度PRO测量工具)
  • 提供或引用PRO测量工具信效度相关证据
  • 明确说明处理缺失数据的统计方法
  • 讨论PRO相关的局限性及其对推广性和临床实践的影响

二、CONSORT-PROs扩展版条目清单

1  CONSORT-PROs扩展版条目清单的中英文对照

内容条目 序号标准清单内容PRO扩展版内容
Title and abstract 标题与摘要1aIdentification as a randomized trial in the title
在题目中体现随机化试验
 
1bStructured summary of trial design, methods, results, and conclusions (for specific guidance see CONSORT for abstracts)
结构化摘要,包括试验设计、方法、结果和结论(详见CONSORT摘要)
P1b. The PRO should be identified in the abstract as a primary or secondary outcome
P1b. 摘要中列出作为主要或次要结局指标的PRO
Introduction
引言
Background and objectives
背景和目的
2aScientific background and explanation of rationale
研究的科学背景和试验的理由
Including background and rationale for PRO assessment
包括PRO作为评价指标的背景和理由
2bSpecific objectives or hypotheses
研究目的或假设
P2b.The PRO hypothesis should be stated and relevant domains identified, if applicable
P2b. 如适用,陈述PRO相关假设及相关维度(如使用多维度PRO测量工具)
Methods
方法
Trial design
试验设计
3aDescription of trial design (such as parallel, factorial), including allocation ratio
试验设计(如平行、析因设计),包括分配比
 
3bImportant changes to methods after trial commencement ( such as eligibility criteria ), with reasons
试验开始后方法上的重要改变(如研究对象选标准的改变)及原因
 
Participants
研究对象
4aEligibility criteria for participants
研究对象的纳入排除标准
Not PRO-specific, unless the PROs were used in eligibility or stratification criteria
通常不需要考虑PRO,除非PROs作为纳入或分层标准
4bSettings and locations where the data were collected
数据收集的机构和地点
 
Interventions
干预
5The interventions for each group with sufficient details to allow replication, including how and when they were actually administered
各组干预的详细内容,包括何时、如何实际开展,以便能够重复
 
Outcomes
结局
6aCompletely defined prespecified primary and secondary outcome measures, including how and when they were assessed
完整明确地定义预先规定的主要和次要结局指标,包括何时、如何评价
P6a. Evidence of PRO instrument validity and reliability should be provided or cited if available including the person completing the PRO and methods of data collection ( paper, telephone, electronic, other)
P6a. 提供或引用PRO测量工具信效度相关证据,包括完成PRO的人员和数据收集的方法(纸质、电话、电子版或其他形式)
6bAny changes to trial outcomes after the trial commenced, with reasons
试验开始后结局的改变及原因
 
Sample size
样本量
7aHow sample size was determined
样本量如何确定
Not required for PRO unless it is a primary study outcome
通常不需要考虑PRO,除非PRO作为主要结局指标
7bWhen applicable, explanation of any interim analyses and stopping guidelines
对期中分析和中止试验的条件进行解释(如适用)
 
Randomization
随机化
Sequence generation
序列产生
8aMethod used to generate the random allocation sequence
产生随机分配序列的方法
 
8bType of randomization; details of any restriction ( such as blocking and block size )
随机化类型;任何限定情况(如区组和区组大小)
 
Allocation concealment mechanism
分配隐藏机制
9Mechanism used to implement the random allocation sequence( such as sequentially numbered containers), describing any steps taken to conceal the sequence until interventions were assigned
实施随机序列的方法(如连续编码的容器),阐明隐藏分配序列的措施
 
Implementation
实施
10Who generated the random allocation sequence, who enrolled participants , and who assigned participants to interventions
产生分配序列、纳入研究对象、分配研究对象的人员
 
Blinding
盲法
11aIf done, who was blinded after assignment to interventions ( for example, participants, care providers, those assessing outcomes) and how
如果实施了盲法,应说明对谁设盲(如研究对象、干预提供者、结局评价者),如何实施的
 
11bIf relevant, description of the similarity of interventions
组间干预的相似性
 
Statistical methods
统计方法
12aStatistical methods used to compare groups for primary and secondary outcomes
组间比较,主要结局与次要结局的统计方法
P12a. Statistical approaches for dealing with missing data are explicitly stated
P12a. 明确说明处理缺失数据的统计方法
12bMethods for additional analyses, such as subgroup analyses and adjusted analyses
其他分析方法,如亚组分析和校正分析
 
Results
结果
Participant flow (a diagram is strongly recommended)
研究对象纳入流程(推荐流程图)
13aFor each group, the numbers of participants who were randomly assigned, received intended treatment, and were analyzed for the primary outcome
各组接受随机分配、接受干预和进入主要结局分析的研究对象数量
The number of PRO outcome data at baseline and at subsequent time points should be made transparent
应清楚报告在基线和随后时间节点PRO相关例数
13bFor each group, losses and exclusions after randomization,together with reasons
各组随机化之后发生的脱落或失访、排除,以及原因
 
Recruitment
研究对象的招募
14aDates defining the periods of recruitment and follow-up
招募研究对象和随访的日期范围
 
14bWhy the trial ended or was stopped
研究结束或停止的原因
 
Baseline data
基线数据
15A table showing baseline demographic and clinical characteristics for each group
反映各组基线人口学特征和临床特征的表格
Including baseline PRO data when collected
收集PRO基线资料
Number analyzed 分析数量16For each group, number of participants (denominator) included in each analysis and whether the analysis was by original assigned groups
各组纳入分析的研究对象数量(分母),是否按照最初分组进行分析
Required for PRO results
有PRO相关结果时均需要描述
Outcomes and estimation
结局和效应估计
17aFor each primary and secondary outcome, results for each group, and the estimated effect size and its precision (such as 95% confidence interval)
对每个主要和次要结局,报告各组结果、效应估计和精度(如95%置信区间)
For multidimensional PRO results from each domain and time point
对于多维度的PRO结果,要列出每个维度和每个时间点的数据
17bFor binary outcomes, presentation of both absolute and relative effect sizes is recommended
对二分类结局,报告绝对效应和相对效应
 
Ancillary analyses
其他分析
18Results of any other analyses performed, including subgroup analyses and adjusted analyses,distinguishing prespecified from exploratory
报告其他分析(包括亚组分析和校正分析)结果,区分预先设定的分析和探索性分析
Including PRO analyses, where relevant
在PRO相关时,包括对PRO数据的分析
Harms
危害
19All important harms or unintended effects in each group
所有重要损害或未预期到的效应
 
Discussion
讨论
Limitations
局限性
20Trial limitations; addressing sources of potential bias; imprecision; and, if relevant, multiplicity of analyses
试验局限性;关注偏倚的来源;不精确程度;多重比较问题
P20/21. PRO-specific limitations and implications for generalizability and clinical practice
P20/21. 讨论PRO相关的局限性及其对外推和临床实践的影响
Generalizability
外推性
21Generalizability (external validity, applicability) of the trial findings
试验结果的外推性(外部有效性、适用性)
Interpretation
结果解释
22Interpretation consistent with results, balancing benefits and harms, and considering other relevant evidence
权衡收益和损害,并考虑其他相关证据,对结果进行解释
PRO data should be interpreted in relation to clinical outcomes including survival data, where relevant
PRO结果的解读需考虑相关的临床结局(如生存资料等)
Other information
其他信息
Registration
注册
23Registration number and name of trial registry
注册机构与注册号
 
Protocol
研究方案
24Where the full trial protocol can be accessed, if available
可以获得完整研究方案的地方 (如适用)
 
Funding
资助
25Sources of funding and other support(such as supply of drugs), role of funders
资助来源和其他支持,资助者的作用
 

三、CONSORT-PROs扩展版使用注意事项

CONSORT-PROs旨在提高RCT中以PRO为主要结局指标或关键的次要结局指标的透明度,以便全面理解涉及PROs的RCT数据的局限性和潜在偏倚来源。作者、审稿专家和读者可联合CONSORT-PROs与CONSORT 2010声明及其他解释和扩展版本使用,从而提高研究报告质量或更好地评价报告。值得注意的是,CONSORT-PROs适用于评估PROs相关结果的报告质量,而非对PROs测量工具质量的评价标准。CONSORT-PROs评估显示研究的报告质量欠佳,并不代表研究结果本身无效、研究设计本身不严谨或研究者未很好地开展研究,但报告质量的欠佳会影响对研究结果的解读。

除了规范报告试验外,研究者也应努力提高试验设计的质量,在试验设计时可参考美国FDA发布的PROs试验相关指南(https://www.bmj.com/content/340/bmj.c2921)和RCT设计相关推荐意见,如干预试验的推荐意见(Recommendations for Interventional Trials Initiative,SPIRIT,https://pubmed.ncbi.nlm.nih.gov/23295957/)。

注:本文内容是参考相关文献后对CONSORT-PROs扩展版报告规范原文的概述,仅代表本网站观点。关于CONSORT-PRO扩展版的更多内容详见官方网站(http://www.consort-statement.org)或Melanie Calvert等发表的论文”Reporting of Patient-Reported Outcomes in Randomized Trials The CONSORT PRO Extension(https://jamanetwork.com/journals/jama/fullarticle/1656259)“、Michael Brundage等发表的论文”Patient-reported outcomes in randomized clinical trials: development of ISOQOL reporting standards(https://pubmed.ncbi.nlm.nih.gov/22987144/)“、成磊等发表的论文”患者报告结局相关随机对照试验的报告规范扩展声明与解读(https://fsjx.cbpt.cnki.net/WKD2/WebPublication/paperDigest.aspx?paperID=60e30e1e-6ddb-41a8-964b-a5f12bdf5b07)“、陈泽等发表的论文”随机对照试验中的患者报告结局:CONSORT PRO 扩展版(http://www.cjebm.com/article/10.7507/1672-2531.202008183)“。

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