Association of low-dose intra-arterial plus intravenous tirofiban with improved functional outcome in branch atheromatous disease-related stroke patients with early neurological deterioration

BACKGROUND: Management of early neurological deterioration (END) in branch atheromatous disease (BAD)-related stroke remains challenging, and intravenous (IV) tirofiban's efficacy is limited. This study aimed to evaluate the efficacy and safety of low-dose intra-arterial (IA) combined with IV tirofiban in BAD-related stroke patients experiencing END. METHODS: We retrospectively analyzed patients with BAD-related stroke who experienced END and were treated with tirofiban between May 2020 and December 2024. Based on the administration route of tirofiban, patients were divided into IA + IV (n = 45) or IV (n = 119) groups. The primary efficacy outcome was the proportion with an excellent functional outcome (modified Rankin Scale [mRS] score 0-1) at 90 days. Secondary outcomes included favorable functional outcome (mRS 0-2), National Institutes of Health Stroke Scale (NIHSS) score at multiple timepoints, and early neurological improvement (ENI). Safety outcomes comprised symptomatic intracranial hemorrhage (sICH), any ICH, mortality, and other serious adverse events. RESULTS: After adjustment for four prespecified prognostic factors, the IA + IV group had significantly higher rate of excellent functional outcome compared to the IV group (adjusted odds ratio [OR], 2.99; 95% CI, 1.25-7.16; P = 0.014). This finding was consistent after IPTW adjustment using 10 covariates (OR, 3.68; 95% CI, 1.48-9.14; P = 0.005). The IA + IV group also exhibited significantly lower NIHSS scores at 24 and 72 h and a higher rate of ENI (75.6% vs. 42.9%; adjusted OR, 4.12; 95% CI, 1.85-9.17; P < 0.001). No sICH occurred in either group. The incidence of any ICH (2.2% vs. 0.8%, P = 0.537) and 90-day all-cause mortality (0% vs. 1.7%, P = 0.997) did not differ significantly between IA + IV and IV groups. CONCLUSIONS: In patients with BAD-related stroke experiencing END, adjunctive low-dose IA + IV tirofiban seems associated with higher odds of excellent functional outcome and faster early neurological recovery compared to IV tirofiban alone. The incidence of safety-related adverse events in this study was low; however, due to limited statistical power, the true safety profile requires further validation. Given the limitations of non-randomized design, procedural confounding, and selection bias, further high-level clinical studies are warranted to validate these findings.