Patients and relatives’ point of view on the choice of outcomes for a randomized-controlled trial in moderate-to-severe Traumatic Brain Injury

Abstract

Traumatic Brain Injury (TBI) is a major cause of mortality and disability worldwide. Clinical research is a cornerstone to improve patients’ outcomes. The empowerment of patients and relatives in research initiatives is now recommended to improve the relevance of trials. We performed an online survey in collaboration with the French National Association of Patients with Brain Injury (France Cérébrolésion) from November to December 2024, to understand their point of view regarding potential outcomes that could qualify hypertonic saline perfusion as relevant in the coming multicentric randomized-controlled COSMOS trial involving 760 moderate-to-severe TBI. 32 persons responded (19 (59.4%) relatives and 13 (40.6%) former patients). Using a 5-grade Likert scale, 23 (71.9%) responders rather agreed or entirely agreed that mortality was a relevant outcome; 26 (81.3%) rated activities of daily living as relevant; 27 (84.4%) rated cognitive function as relevant; 30 (93.7%) rated memory function as relevant; 17 (53.2%) rated functional outcome as relevant; 26 (81.3%) rated anxiety and depression symptoms as relevant; 27 (84.4%) rated quality of life as relevant. Ten (31.3%) responders could not understand the concept of functional outcome. The highest-ranked outcomes were mortality (16 responders gave the highest mark of 7), activities in daily living (14 responders), and memory (11 responders). Although this survey does not have the value of a consensus and further studies are needed to encompass other stakeholders’ opinions, we have modified the primary outcomes in COSMOS: functional outcome has been replaced by a hierarchy with 1/ survival and 2/activities in daily living.