Efficacy of Cerebrolysin Treatment as an Add-On Therapy to Mechanical Thrombectomy in Patients with Acute Ischemic Stroke Due to Large Vessel Occlusion in Anterior Circulation: Results of a 3-Month Follow-up of a Prospective, Open Label, Single-Cente

This study hypothesized that Cerebrolysin, a multimodal neuroprotective agent, enhances the efficacy and safety of mechanical thrombectomy (MT) in both acute ischemic stroke (AIS) and recovery stroke phases in selected patients with good collateral status (CTA-CS 2-3) and effective recanalization (mTICI 2b-3). A single-center, prospective, open-label, single-arm study with blinded outcome assessment of 50 consecutive patients with moderate-to-severe AIS treated with MT ≤ 6 h of stroke onset followed by Cerebrolysin (30 ml iv within 8 h of onset and continued to day 21, first cycle) and in a recovery phase (between 69-90 days, second cycle) compared to 50 historical controls matched by propensity scores. Key outcomes included functional independence (mRS 0-2 at 90 days), safety endpoints, and neurological recovery (NIHSS at 24 h and 7 day post MT). Patients receiving Cerebrolysin achieved higher rates of mRS 0-2 at 90 days (68% vs. 44%, p = 0.016, OR 2.7, 95% CI 1.2-6.1; NNT: 4.2), had reduced risk of secondary ICH (14% vs. 40%, p = 0.02; RR 0.37, 95% CI 0.14-0.95), and had lower NIHSS on day 7 (median [IQR]: 3 [4] vs. 6 [9], p = 0.01). There was a significant difference in Barthel Index scores between the Cerebrolysin group and the control group at 30 days (median [IQR]: 77 [32] vs. 63 [50], p = 0.03) and at 3 months (86 [22] vs. 75 [29], p = 0.01) primarily driven by the increase in the mobility and transfer components. Multivariate analysis identified Cerebrolysin as an independent predictor of favorable outcomes at 3 months (OR 7.5, 95% CI 1.8-30.9), particularly in patients with diabetes (interaction OR 9.6, 95% CI 1.01-92). The overall mortality rates at 30 and 90 days were similar in both groups (2% vs 6% and 8% vs 12%, p > 0.1). Cerebrolysin improved functional outcomes at 90 days, accelerated neurological recovery, and reduced complications post-MT in patients with small ischemic core, good collateral circulation, and effective recanalization at baseline. These findings warrant further randomized trials to validate its efficacy and explore its long-term benefits.Registration: URL: https://www.clinicaltrials.gov ; unique identifier: NCT04904341.