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Abstract：
Over the last six decades (earliest included publication from 1959), clinical trials of migraine preventive treatments have led to the regulatory approval of many medications and devices. Despite similar clinical goals, the outcomes and endpoints used in these trials are broad and not well standardized.
To describe results from a systematic literature review focused on outcomes and endpoints used in preventive migraine clinical trials.
A systematic literature review, following a pre-specified (unregistered) protocol developed to adhere to recommendations of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses, was conducted to characterize the endpoints and outcomes used in preventive migraine clinical trials. Predetermined terms were searched in PubMed on October 28, 2019. Data related to trial design, subject characteristics, outcomes, and endpoints reported in each publication were extracted. Descriptive summaries of these features were tabulated for the recent subset of publications, published during or after 1988, that were randomized, blinded, and focused on pharmacological or device therapies for the preventive treatment of migraine.
The initial literature search identified 1506 publications, of which 757 publications were eligible for data extraction. Of specific clinical interest were the recent subset of 268 articles (268/757, 35.4%) fulfilling the targeted criteria. Results showed that the outcomes used to define endpoints varied substantially across publications. For example, in the recent subset of publications, 68.7% (184/268) of the publications examined ≥1 migraine-specific outcome, 39.6% (106/268) examined ≥1 headache-specific outcome, 50.7% (136/268) examined ≥1 acute/rescue medication use outcome, 40.3% (108/268) examined ≥1 headache-related patient-reported outcome measure (PROM), and 22.0% (59/268) examined ≥1 non-headache-specific PROM. Furthermore, the definition of the endpoints used (e.g., change from baseline, fixed timepoint comparisons, categorization of \"responders\" to treatment based on wide variety of \"responder definitions\") also differed across publications.
Publications from clinical trials of preventive migraine pharmacologic and device treatments differed in terms of study design, endpoint definitions, and how endpoints and outcomes were measured. Although there were common outcomes and endpoints used across publications, no clear \"standardized\" set of endpoints and outcomes emerged. The inconsistencies in endpoints and outcomes within this literature suggest that the development of a uniform set of outcomes and endpoints could improve the clinical meaningfulness of clinical trial results, facilitate cross-trial comparisons and better inform patient care. This standard set of outcomes and endpoints should be statistically robust and informed by the priorities of various stakeholders, most importantly, the needs and preferences of people living with migraine.
To describe results from a systematic literature review focused on outcomes and endpoints used in preventive migraine clinical trials.
A systematic literature review, following a pre-specified (unregistered) protocol developed to adhere to recommendations of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses, was conducted to characterize the endpoints and outcomes used in preventive migraine clinical trials. Predetermined terms were searched in PubMed on October 28, 2019. Data related to trial design, subject characteristics, outcomes, and endpoints reported in each publication were extracted. Descriptive summaries of these features were tabulated for the recent subset of publications, published during or after 1988, that were randomized, blinded, and focused on pharmacological or device therapies for the preventive treatment of migraine.
The initial literature search identified 1506 publications, of which 757 publications were eligible for data extraction. Of specific clinical interest were the recent subset of 268 articles (268/757, 35.4%) fulfilling the targeted criteria. Results showed that the outcomes used to define endpoints varied substantially across publications. For example, in the recent subset of publications, 68.7% (184/268) of the publications examined ≥1 migraine-specific outcome, 39.6% (106/268) examined ≥1 headache-specific outcome, 50.7% (136/268) examined ≥1 acute/rescue medication use outcome, 40.3% (108/268) examined ≥1 headache-related patient-reported outcome measure (PROM), and 22.0% (59/268) examined ≥1 non-headache-specific PROM. Furthermore, the definition of the endpoints used (e.g., change from baseline, fixed timepoint comparisons, categorization of \"responders\" to treatment based on wide variety of \"responder definitions\") also differed across publications.
Publications from clinical trials of preventive migraine pharmacologic and device treatments differed in terms of study design, endpoint definitions, and how endpoints and outcomes were measured. Although there were common outcomes and endpoints used across publications, no clear \"standardized\" set of endpoints and outcomes emerged. The inconsistencies in endpoints and outcomes within this literature suggest that the development of a uniform set of outcomes and endpoints could improve the clinical meaningfulness of clinical trial results, facilitate cross-trial comparisons and better inform patient care. This standard set of outcomes and endpoints should be statistically robust and informed by the priorities of various stakeholders, most importantly, the needs and preferences of people living with migraine.
摘要：
在过去的六十年中（最早包括1959年的出版物），偏头痛预防性治疗的临床试验已导致许多药物和设备获得监管部门的批准。尽管临床目标相似，这些试验中使用的结局和终点是广泛的,并且没有很好的标准化.
描述系统性文献综述的结果，重点是预防性偏头痛临床试验中使用的结果和终点。
系统的文献综述，遵循预先指定的（未注册）协议，以遵守系统审查和荟萃分析的首选报告项目的建议，进行表征用于预防性偏头痛临床试验的终点和结局。预定术语于2019年10月28日在PubMed中搜索。与试验设计相关的数据，主题特征,结果，并提取各出版物中报告的终点。这些功能的描述性摘要已针对最近的出版物子集列出，在1988年或之后发表的，是随机的，失明,并专注于预防性治疗偏头痛的药物或设备疗法。
最初的文献检索确定了1506种出版物，其中757份出版物符合数据提取条件。具有特定临床兴趣的是符合目标标准的268篇文章的最新子集（268/757，35.4%）。结果表明，用于定义终点的结果在出版物中差异很大。例如,在最近的出版物中，68.7％（184/268）的出版物检查≥1偏头痛特异性结局，39.6％（106/268）检查≥1头痛特异性结局，50.7％（136/268）检查≥1急性/抢救药物使用结果，40.3%（108/268）检查≥1例头痛相关患者报告结果测量（PROM），22.0%(59/268)检查≥1例非头痛特异性PROM。此外，使用的端点的定义(例如，从基线变化，固定时间点比较，根据各种“响应者定义”)对治疗的“响应者”分类也不同。
预防性偏头痛药物和器械治疗临床试验的出版物在研究设计方面有所不同，端点定义,以及如何测量终点和结果。尽管在出版物中使用了共同的结果和终点，没有出现明确的“标准化”终点和结果集。本文献中终点和结果的不一致表明，开发一组统一的结果和终点可以提高临床试验结果的临床意义。促进交叉试验比较并更好地告知患者护理.这套标准的结果和终点应该在统计上是稳健的，并以各利益攸关方的优先事项为依据。最重要的是,偏头痛患者的需求和偏好。
描述系统性文献综述的结果，重点是预防性偏头痛临床试验中使用的结果和终点。
系统的文献综述，遵循预先指定的（未注册）协议，以遵守系统审查和荟萃分析的首选报告项目的建议，进行表征用于预防性偏头痛临床试验的终点和结局。预定术语于2019年10月28日在PubMed中搜索。与试验设计相关的数据，主题特征,结果，并提取各出版物中报告的终点。这些功能的描述性摘要已针对最近的出版物子集列出，在1988年或之后发表的，是随机的，失明,并专注于预防性治疗偏头痛的药物或设备疗法。
最初的文献检索确定了1506种出版物，其中757份出版物符合数据提取条件。具有特定临床兴趣的是符合目标标准的268篇文章的最新子集（268/757，35.4%）。结果表明，用于定义终点的结果在出版物中差异很大。例如,在最近的出版物中，68.7％（184/268）的出版物检查≥1偏头痛特异性结局，39.6％（106/268）检查≥1头痛特异性结局，50.7％（136/268）检查≥1急性/抢救药物使用结果，40.3%（108/268）检查≥1例头痛相关患者报告结果测量（PROM），22.0%(59/268)检查≥1例非头痛特异性PROM。此外，使用的端点的定义(例如，从基线变化，固定时间点比较，根据各种“响应者定义”)对治疗的“响应者”分类也不同。
预防性偏头痛药物和器械治疗临床试验的出版物在研究设计方面有所不同，端点定义,以及如何测量终点和结果。尽管在出版物中使用了共同的结果和终点，没有出现明确的“标准化”终点和结果集。本文献中终点和结果的不一致表明，开发一组统一的结果和终点可以提高临床试验结果的临床意义。促进交叉试验比较并更好地告知患者护理.这套标准的结果和终点应该在统计上是稳健的，并以各利益攸关方的优先事项为依据。最重要的是,偏头痛患者的需求和偏好。
