Effect of IL-6 receptor inhibition on infarct volume after endovascular treatment for ischaemic stroke: a phase 2, randomised, placebo-controlled trial.

backgroundTocilizumab, an interleukin-6 receptor inhibitor, is a promising cytoprotective agent selected by the Stroke Preclinical Assessment Network. It showed a protective effect on infarct volume and functional outcomes in animal stroke models.

methodsIn this investigator-initiated, multicentre, randomised, double-blind, placebo-controlled trial, patients with acute ischaemic stroke undergoing EVT were recruited. Eligible patients were randomly assigned (1:1) to receive tocilizumab or placebo treatment. Both patients and investigators were blinded to the treatment assignments. A single dose of tocilizumab (240 mg) or placebo was administered intravenously as soon as possible within 24 h after stroke onset and within 1 h after randomisation. The primary efficacy outcome was the change in infarct volume from baseline (before EVT and start of study drug) to 72 h. Primary and safety analyses were done in the intention-to-treat population. This trial is registered with ClinicalTrials.gov, NCT06238024.

findingsA total of 108 patients were enrolled (n placebo = 57; n tocilizumab = 51). The median change in infarct core volume between baseline and 72 h was 27.0 mL (7.6-62.4) in the placebo group and 8.8 mL (IQR 3.4-20.6) in the tocilizumab group (adjusted mean difference in cubic root volume [ml

interpretationAmong patients with acute ischaemic stroke undergoing endovascular treatment, tocilizumab tended to reduce infarct volume growth at 72 h post-treatment and is well tolerated. Future trials are necessary to confirm the beneficial effect of tocilizumab on long-term functional outcome following stroke.

fundingNoncommunicable Chronic Diseases-National Science and Technology Major Project, Beijing Nova Program, National Natural Science Foundation of China, Beijing Natural Science Foundation.