Clinical and Safety Outcomes of Edaravone Dexborneol in Acute Ischemic Stroke

BACKGROUND AND OBJECTIVES: Although edaravone dexborneol, a multitarget cytoprotective drug, has demonstrated benefits in previous clinical trials, its actual clinical efficacy and safety in patients with acute ischemic stroke (AIS) remain unclear. This study aimed to test the hypothesis that edaravone dexborneol is associated with better outcomes in patients with AIS in real-world clinical practice. METHODS: We conducted a prospective, multicenter, real-world cohort study at 72 centers in China from January 14 to July 4, 2023. We included patients age 18 years or older, with AIS within 14 days of onset, and a prestroke modified Rankin Scale (mRS) of 0 or 1. Patients were divided into those receiving edaravone dexborneol (the exposed group) or not (the unexposed group). Clinical outcomes included favorable functional outcome (mRS score 0-1) at 90 days, symptomatic intracerebral hemorrhage (sICH) during the hospital, and all-cause mortality within 90 days. Multivariable logistic regression, propensity score matching (PSM), and inverse probability of treatment weighting (IPTW) analyses were conducted. RESULTS: Of 4,401 participants (2,904 men [66.8%]; median [interquartile range] age, 65 [57-72] years), 3,017 (68.6%) were treated with edaravone dexborneol. The exposed group was younger (65 [56-72] years vs 66 [58-74] years), had higher NIH Stroke Scale (3 [2-7] vs 3 [1-5]), and had shorter time from onset to admission (7.0 [2.5-24.0] hours vs 10.4 [2.8-48.0] hours). The exposed group had a higher proportion of patients with favorable functional outcome at 90 days compared with the unexposed group (68.6% [2,071/3,017] vs 66.0% [914/1,384], adjusted odds ratio [aOR] 1.23 [95% CI 1.06-1.43]). Moreover, sICH rates (0.4% vs 0.6%, aOR 0.44 [95% CI 0.14-1.44]) and mortality within 90 days (2.1% vs 3.3%, adjusted hazard ratio 0.89 [95% CI 0.58-1.37]) were similar in both groups. PSM and IPTW analyses yielded results consistent with the multivariate adjustment model. DISCUSSION: In this observational cohort study involving Chinese patients with AIS, edaravone dexborneol was associated with significantly better functional outcome at 90 days. Verification of our findings is warranted in other populations. TRIAL REGISTRATION INFORMATION: This trial is registered with Effectiveness and Safety of Edaravone Dexborneol in Acute Ischemic Stroke, number NCT05644223; submitted for registration on November 30, 2022; first patient enrollment was on January 14, 2023. CLASSIFICATION OF EVIDENCE: This study provides Class III evidence that treatment of patients with AIS with edaravone dexborneol is associated with significantly better functional outcome at 90 days measured using the mRS.