Systematic review of outcomes and endpoints in preventive migraine clinical trials.
Systematic review of outcomes and endpoints in preventive migraine clinical trials.
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作者：
McGinley JS;Houts CR;Nishida TK;Buse DC;Lipton RB;Goadsby PJ;Dodick DW;Wirth RJ
在过去的60年里(最早包括1959年发表的文章)，偏头痛预防治疗的临床试验导致了许多药物和设备的监管批准。尽管临床目标相似，但在这些试验中使用的结果和终点是广泛的，并且没有很好地标准化。描述一项系统的文献综述的结果，重点是在预防性偏头痛临床试验中使用的结果和终点。按照系统综述和荟萃分析首选报告项目的建议，遵循预先指定(未注册)的方案，进行了一项系统的文献综述，以确定预防性偏头痛临床试验中使用的终点和结果。2019年10月28日，在PubMed上搜索了预定的术语。摘录了与试验设计、受试者特征、结果和终点相关的数据，这些数据在每个出版物中都有报道。这些特征的描述性摘要被列成表格，用于1988年期间或之后出版的最近的出版物子集，这些出版物是随机、盲目的，重点是用于偏头痛预防治疗的药物或设备疗法。最初的文献检索确定了1506种出版物，其中757种出版物符合数据摘录条件。临床上特别感兴趣的是最近符合目标标准的268篇文章的子集(268/757，35.4%)。结果表明，用于定义终点的结果在不同的出版物中有很大的不同。例如，在最近的出版物中，68.7%(184/268)研究了≥1偏头痛特有的结果，39.6%(106/268)研究了≥1头痛特有的结局，50.7%(136/268)研究了≥1急性/抢救药物使用结果，40.3%(108/268)研究了≥1头痛相关患者报告结局测量(PROM)，22.0%(59/268)研究了≥1非头痛特异性胎膜早破。此外，所用终点的定义(例如，从基线的改变、固定时间点的比较、根据各种“应答者定义”对“应答者”的分类到治疗)也因出版物而异。偏头痛预防性药理学和设备治疗的临床试验的出版物在研究设计、终点定义以及终点和结果的测量方式方面存在差异。尽管各出版物使用了共同的结果和终点，但没有出现明确的“标准化”终点和终点。这篇文献中终点和结果的不一致表明，制定一套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，并更好地为患者护理提供信息。这套标准的结果和终点在统计上应该是稳健的，并根据不同利益攸关方的优先事项提供信息，最重要的是，偏头痛患者的需求和偏好。
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.
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