Effectiveness and safety of edaravone dexborneol in acute ischemic stroke (EXPAND): Study protocol for a prospective, multicenter, real-world cohort study

Abstract

Background: Edaravone dexborneol has been confirmed as a novel neuroprotective agent by acting on multiple active targets of antioxidation and anti-inflammation. Objectives: This study aims to evaluate the effectiveness and safety of edaravone dexborneol in patients with acute ischemic stroke (AIS) within 14 days of symptom onset in real-world clinical practice. Methods: This prospective, multicenter, 2-arm, parallel, real-world cohort study will involve 72 stroke centers in China and plans to recruit 4750 exposed/unexposed patients with a 2:1 ratio to gather additional safety information on edaravone dexborneol. Patients eligible of the Effectiveness and Safety of Edaravone Dexborneol in Acute Ischemic Stroke (EXPAND) are diagnosed with AIS within 14 days of symptom onset. The eligible patients who receive edaravone dexborneol will be assigned to the exposed group, while those without using edaravone dexborneol will be assigned to the unexposed group. All participants will be followed up for 90 days. The primary outcome is a favorable functional outcome as defined by a modified Rankin Scale (mRS) score 0–1 at 90 days. Primary efficacy in the 2 groups will be compared using multivariable logistic regression. Results: EXPAND is the first real-world study to elucidate the efficacy and safety of edaravone dexbornel in AIS, which has the potential to extend the therapeutic time window of neuroprotective treatment and propose a multiple-target approach for AIS. Conclusions: The protocol was approved by the Ethics Committee at all participating sites [the reference number of central ethic committee: (2022)090] and by the local Institutional Review Board of each participating center. The findings of the study will be published in peer-reviewed scientific journals and presented at national and international conferences. This cohort study only collects real-world patient data during the diagnosis and treatment process. The refusal of a patient to participate or the decision to withdraw from the study will not interfere with the clinical decision-making. Thus, no additional risk will be imposed. Trial registration number: NCT05644223.