Utilization and Reporting of Patient-Reported Outcome Measures in Randomized Clinical Trials of Acute Stroke (2010-2020).

BACKGROUND

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.

METHODS

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.

RESULTS

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.

CONCLUSIONS

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.

REGISTRATION

URL: https://www.crd.york.ac.uk/PROSPERO/; Unique identifier: CRD42019128727.