Standardized Outcomes for Randomized Controlled Trials Targeting Early Interventions in Patients With Moderate-to-Severe Traumatic Brain Injury: Protocol for the Development of a Core Outcome Set (Preprint)
Authors: Cinotti Raphael , Derouin Yvan , Chenet Amandine , Oujamaa Lydia , Glize Bertrand , Launey Yoann , Dahyot-Fizelier Claire , Cartron Emmanuelle , Renvoise Melodie , Sautenet Benedicte , Sebille Veronique 
Published: November 2023

BACKGROUND

<i>:</i> With more than 60 million new cases around the world each year, traumatic brain injury (TBI) causes substantial mortality and morbidity. Managing TBI is a major human, social, and economic concern. In the last 20 years, there has been an increase in clinical trials in neurocritical care, leading mostly to negative results. The evaluation of neurological outcomes, predominantly as primary outcomes, using clinical scales (Glasgow Outcome Scale) has limitations that could explain these results. Moreover, patient-centered outcomes are seldom reported despite their recognized clinical relevance.

OBJECTIVE

<i>:</i> The aim of this project is to establish a core outcome set (COS) for patients with moderate-to-severe TBI in randomized control trials in neurocritical care research.

METHODS

This study will follow five distinct steps: (1) systematic review to identify outcomes that have been reported in trials; (2) semistructured interviews with patients and their families to identify their priorities after TBI and explore potential patient-centered outcomes; (3) health care stakeholder focus groups with clinicians, researchers, and policy makers to describe potential outcomes; (4) an eDelphi survey with stakeholder groups to make a list of previously identified core outcomes; and (5) a consensus workshop to establish a COS for moderate-to-severe TBI clinical trials.

RESULTS

<i>:</i> The systematic review was published in August 2024. Regarding Step 2, 30 semistructured interviews of patients and relatives were performed from July 2021 to December 2023, and analyses were completed in October 2024. Step 3 is currently under development, and Step 4 is planned for the end of 2025. Step 5 is expected to occur during fall/winter 2026.
<strong>Conclusions</strong>: Establishing a COS, to be consistently measured and reported in TBI trials in neurocritical care will ensure rigorous reporting, avoid bias, and improve the integrity, transparency, and usability of clinical research. The French context of the study is the main limitation, but we are seeking international collaboration on the project. The results of each step of the project will be disseminated through abstracts, publications, and patient associations.

CONCLUSIONS

Establishing a COS, to be consistently measured and reported in TBI trials in neurocritical care will ensure rigorous reporting, avoid bias, and improve the integrity, transparency, and usability of clinical research. The French context of the study is the main limitation, but we are seeking international collaboration on the project. The results of each step of the project will be disseminated through abstracts, publications, and patient associations.

INTERNATIONAL REGISTERED REPORT

DERR1-10.2196/54525