To evaluate the safety and efficacy of Edaravone-dexborneol in patients with AIS by synthesizing evidence from randomized controlled trials and observational studies.

Each year, approximately 15 million people suffer an acute ischemic stroke worldwide, of which 5 million suffer death and another 5 million are left with long-term disability. Existing treatments exhibit limited efficacy, warranting the investigation of novel therapeutic strategies. Edaravone and Dexborneol have surfaced as potential treatments for AIS, showing promise in facilitating neurological recovery and mobility.

The findings indicate that the combination of Edaravone-dexborneol decreases the infarct size, oxidative stress, and inflammation in ischemic brain regions. RCTs demonstrated significant improvements in 90-day functional recovery with Edaravone-dexborneol compared with Edaravone alone or standard therapy. One multicenter phase III RCT reported favorable mRS outcomes in 67.2% of patients receiving Edaravone-dexborneol versus 59.0% with Edaravone alone (OR 1.42, 95% CI 1.12–1.81; p=0.004). Another phase II trial found 70.7% of patients achieved mRS <2 versus 47.8% with standard therapy (p=0.031). Across studies, serious adverse events were infrequent, and the safety profile was comparable to existing treatments.

This is among the first reviews synthesizing clinical trial evidence on Edaravone-dexborneol, by filling in the translational gap and exhibiting the safety and efficacy of this neuroprotective adjunct in improving outcomes in AIS. Current evidence is largely limited to Asian populations with short follow-up periods. Larger multicenter trials across diverse populations are needed to validate long-term efficacy and safety. If validated in diverse populations, this therapy provides neurologists with a neuroprotective adjunct to current AIS treatments and expands treatment options beyond time-limited reperfusion strategies.