Hostnik Joseph S, Strong Brent, Reeves Mathew J
College of Human Medicine (J.S.H.), Michigan State University, East Lansing.
Department of Epidemiology and Biostatistics (B.S., M.J.R.), Michigan State University, East Lansing.
Stroke. 2024 May;55(5):1174-1180. doi: 10.1161/STROKEAHA.123.046209. Epub 2024 Mar 21.
Patient-reported outcome measures (PROMs) describe health status from the perspective of the patient. There is growing interest in incorporating PROMs into clinical trials, but the extent that such measures are used in contemporary stroke trials is uncertain. We sought to determine how often acute stroke trials included PROMs as outcome measures and assessed the completeness of methodological reporting.
We searched MEDLINE for randomized controlled trials published in 9 high-impact journals between 2010 and 2020. Eligible studies were phase 2 or 3 trials that tested therapeutic interventions within 1 month of stroke onset. Using the trial's primary publication and protocol, we abstracted key study characteristics including all primary and secondary outcome measures. We defined PROMs as self-reported measures of quality of life, symptoms, or function collected without interpretation of an external party.
Of 116 trials that met eligibility, 57 (49%) included at least 1 PROM. Of these, 41 trials (35%) included a PROM in its primary publication, while 16 (14%) identified a PROM in its protocol. Only 1 trial used a PROM as a primary outcome. Among the 57 total trials, the most commonly used measures were Euro-QOL (n=41, 72%), Stroke Impact Scale (n=10, 18%), and Short-Form 36 (n=6, 11%). Trials were more likely to include a PROM if they were published after 2016, were phase 3, or included only hemorrhagic stroke. Of the 41 trials that included a PROM in the primary publication, 40 (97%) provided PROM results, but only 9 (22%) found statistically significant differences between treatment groups. Quality of methodological reporting was generally poor.
Half of contemporary acute stroke trials published in high-impact journals listed at least 1 PROM as a secondary outcome, but they played a minor role in the presentation of the final trial results. Inclusion of PROMs in acute stroke trials requires greater attention during both the design and reporting phases of the trial.
URL: https://www.crd.york.ac.uk/PROSPERO/; Unique identifier: CRD42019128727.
患者报告结局测量（PROMs）从患者的角度描述健康状况。将PROMs纳入临床试验的兴趣日益浓厚，但在当代中风试验中使用此类测量的程度尚不确定。我们试图确定急性中风试验将PROMs作为结局测量的频率，并评估方法学报告的完整性。
我们在MEDLINE中检索了2010年至2020年期间在9种高影响力期刊上发表的随机对照试验。符合条件的研究为2期或3期试验，在中风发作后1个月内测试治疗性干预措施。利用试验的主要出版物和方案，我们提取了关键的研究特征，包括所有主要和次要结局测量。我们将PROMs定义为在没有外部方解释的情况下收集的关于生活质量、症状或功能的自我报告测量。
在116项符合条件的试验中，57项（49%）至少纳入了1项PROM。其中，41项试验（35%）在其主要出版物中纳入了一项PROM，而16项（14%）在其方案中确定了一项PROM。只有1项试验将PROM用作主要结局。在总共57项试验中，最常用的测量方法是欧洲生活质量量表（n = 41，72%）、中风影响量表（n = 10，18%）和简短健康调查问卷36项（n = 6，11%）。如果试验在2016年后发表、为3期试验或仅包括出血性中风，则更有可能纳入PROM。在主要出版物中纳入PROM的41项试验中，40项（97%）提供了PROM结果，但只有9项（22%）发现治疗组之间存在统计学上的显著差异。方法学报告的质量普遍较差。
在高影响力期刊上发表的当代急性中风试验中，有一半将至少1项PROM列为次要结局，但它们在最终试验结果的呈现中作用较小。在急性中风试验的设计和报告阶段，都需要更加关注PROM的纳入。
网址：https://www.crd.york.ac.uk/PROSPERO/；唯一标识符：CRD42019128727。
