Could you please provide a summary of the available literature on the use of TNK in extended window stroke? In particular, I would appreciate insights into efficacy vs. safety, as well as any available comparisons between TNK and alteplase in this context. Thank you!

Comment by InpharmD Researcher

The use of tenecteplase in the management of extended window stroke has been a subject of investigation, although direct comparisons with alteplase are limited in this specific clinical scenario. Recent meta-analyses found that intravenous thrombolysis, including tenecteplase, administered beyond 4.5 hours, was associated with statistically significant improvements in functional outcomes, particularly when guided by advanced imaging. Some data revealed mixed results, however, suggesting that use in the extended window results in improvement in some outcomes (e.g., excellent functional outcome), but not necessarily others (e.g., good functional outcome, disability, or mortality). A limited amount of comparative data between tenecteplase and alteplase in this setting demonstrated comparable benefit in the extended window.

Background

A 2025 meta-analysis systematically evaluated the efficacy and safety of intravenous thrombolysis (IVT) administered beyond 4.5 hours of ischemic stroke onset in adult patients who did not undergo mechanical thrombectomy. The analysis included data from eight randomized controlled trials (RCTs), encompassing a pooled cohort of 1,742 patients across 263 centers globally. Selection criteria included RCTs comparing IVT (using either alteplase or tenecteplase [TNK]) with standard medical care or placebo beyond the traditional 4.5-hour time window, with primary endpoints focused on functional outcomes measured by the modified Rankin Scale (mRS 0–1 and mRS 0–2) at 90 days, as well as rates of symptomatic intracerebral hemorrhage (sICH) and all-cause mortality. Patient populations were derived from diverse regions, including China, Europe, and Australia, and eligibility was determined using advanced imaging modalities such as diffusion-weighted imaging–fluid-attenuated inversion recovery (DWI-FLAIR) mismatch or perfusion-based imaging. Of note, three RCTs specifically evaluated TNK (EXIT-BT - last known well to intervention 5.5 (5.2–5.7) hrs, ROSE-TNK - 11.47±4.70 hrs, and TRACE-III - 12.4 (8.8–16.3) hrs), enrolling a total of 706 patients (353 in the TNK group and 353 in the control group; all conducted in China). [1]

The analysis demonstrated that IVT administered beyond 4.5 hours was associated with significantly improved functional outcomes compared to standard medical care. Specifically, patients receiving IVT had higher odds of achieving excellent (mRS 0–1: odds ratio [OR] 1.43, 95% confidence interval [CI] 1.17–1.75; p= 0.0005) and good (mRS 0–2: OR 1.36, 95% CI 1.12–1.66; p= 0.002) recovery at 90 days, with negligible heterogeneity across studies (I² = 0%). However, IVT recipients experienced increased rates of sICH (OR 4.25, 95% CI 1.67–10.84; p= 0.002), although mortality at 90 days did not differ significantly between IVT and control groups (OR 1.28, 95% CI 0.87–1.89; p= 0.21). Subgroup analyses revealed that patients selected with perfusion imaging had marginally higher odds of excellent outcomes (OR 1.45, 95% CI 1.08–1.94) compared to those selected using a DWI-FLAIR mismatch approach (OR 1.34, 95% CI 0.94–1.91). Additionally, subanalysis showed the use of TNK (OR 1.47, 95% CI 1.06–2.04) yielded a comparable benefit to alteplase (OR 1.38, 95% CI 1.08–1.78) in the extended window. These findings underscore the efficacy of imaging-guided IVT beyond 4.5 hours and suggest a potential role for broader thrombolytic use in settings lacking access to endovascular therapy. [1]

A 2024 systematic review and meta-analysis synthesized data from three RCTs evaluating the efficacy and safety of IV TNK 0.25 mg/kg in patients with acute ischemic stroke (AIS) presenting in the extended time window, defined as beyond 4.5 hours from last-seen-well up to 24 hours. The included trials, TWIST, TIMELESS, and ROSE-TNK, enrolled a total of 1,116 patients (556 receiving TNK and 560 controls). The eligibility was based on neuroimaging and clinical selection criteria varying across trials. Primary efficacy was defined as the achievement of an excellent functional outcome (mRS score ≤1) at 3 months. Secondary endpoints encompassed good outcome (mRS ≤2), reduced disability (≥1-point reduction on mRS), sICH, any ICH, and 3-month mortality. A random-effects model was employed to generate pooled estimates, with risk ratios (RR) and common odds ratios (cOR) accompanied by 95% CIs. The meta-analysis demonstrated that TNK administration was associated with a statistically significant improvement in the likelihood of excellent 3-month functional outcome compared to controls (RR 1.17; 95% CI 1.01–1.36; I²=0%). However, the differences for good outcome (RR 1.05; 95% CI 0.94–1.17) and reduced disability (adjusted cOR 1.14; 95% CI 0.92–1.40) were not statistically significant. Importantly, safety outcomes indicated comparable risks between TNK and control groups: sICH (RR 1.67; 95% CI 0.70–4.00), any ICH (RR 1.08; 95% CI 0.90–1.29), and 3-month mortality (RR 1.10; 95% CI 0.81–1.49), with no detected heterogeneity across trials. Subgroup analyses stratified by endovascular therapy (EVT) administration revealed consistent outcomes in reduced disability, regardless of EVT status. Interpretation is supported by a low pooled sICH rate in the TNK group (2%), aligning with previous reports of favorable hemorrhagic profiles for TNK at this dose. These findings reinforce the potential utility of TNK 0.25 mg/kg as a thrombolytic agent in selected AIS patients beyond the standard treatment window, pending further evidence from ongoing phase III trials. [2]

A 2025 systematic review and meta-analysis of randomized clinical trials investigated the efficacy and safety of administering TNK in AIS patients who presented within a 4.5 to 24-hour window from the last known well (LKW) time. The analysis included three RCTs, encompassing a total of 1,054 patients, with 532 receiving TNK and 522 receiving standard medical therapy. The pooled data demonstrated that TNK treatment was associated with a 33% higher likelihood of achieving functional independence (mRS 0–2) at 90 days, as evidenced by an odds ratio of 1.33, with a 95% confidence interval of 1.04–1.70 and a p-value of 0.023. However, no significant difference was observed in the ordinal mRS shift. Safety analysis indicated no statistically significant increase in sICH rates or 90-day mortality among patients treated with TNK compared to standard medical therapy. The analysis further detailed the inclusion criteria and methodologies of the RCTs, highlighting that patient selection varied slightly in terms of pre-stroke mRS scores and imaging criteria, such as computed tomography perfusion (CTP) and magnetic resonance imaging (MRI) with diffusion-weighted imaging (DWI). The trials utilized different definitions for outcome measures, such as early neurological improvement and sICH, contributing to the heterogeneity observed in the pooled results. Despite these variations, the comprehensive data synthesis suggested that TNK might offer an effective treatment option for AIS patients beyond the traditional 4.5-hour window, particularly in settings with limited access to endovascular thrombectomy (EVT). This expansion of the therapeutic window could significantly increase the number of patients eligible for treatment, potentially reducing the global burden of stroke-related disability. However, the analysis also called for larger, well-powered studies to confirm these findings and address the limitations of the current data regarding patient selection and heterogeneity. [3]

A 2021 meta-analysis evaluated the use of IV alteplase in patients who presented with a stroke of unknown time of onset, particularly in those with perfusion-diffusion MRI, perfusion computed tomography (CT), or MRI with DWI-FLAIR mismatch. Results from four randomized controlled trials (N= 843; alteplase n= 429) demonstrated that favourable functional outcome (score of 0-1 on the modified Rankin Scale [mRS] = no disability) at 90 days occurred more in the alteplase group vs. placebo (47% vs. 39%; adjusted odds ratio [aOR] 1.49, 95% confidence interval [CI] 1.10 to 2.03, p= 0.011). Additionally, patients who received alteplase had a significant shift towards a better functional outcome (adjusted common odds ratio 1.38, 95% CI 1.05 to 1.80, p= 0.019) and a higher odds of independent outcome as defined as score 0-2 on the mRS (aOR 1.50, 95% CI 1.06 to 2.12). Numerically, more patients in the placebo group (25%) were severely disabled or dead (mRS 4–6) compared to the alteplase group (21%). Death occurred in 6% patients with alteplase and 3% patients among controls (aOR 2.06, 95% CI 1.03 to 4.09, p= 0.040). Symptomatic intracerebral hemorrhage (sICH) occurred more frequently in alteplase-treated patients than the controls (3% vs. 1%, aOR 5.58, 95% CI 1.22 to 25.50, p= 0.024). Pre-defined subgroup analysis did not reveal a significant heterogeneity of the treatment effect across different variables, including dose of alteplase (0.9 vs. 0.6 mg/kg body weight), age (≤60 vs >60 years), sex, and baseline stroke severity (NIHSS ≤10 vs.>10). On the other hand, patients with a history of stroke and transient ischemic attack experienced a larger benefit with IV alteplase (OR 4.5, 95% CI 1.58 to 12.8, p= 0.02). Although the overall rate of sICH in this analysis was higher compared to the placebo, it was similar to the rate (3.5%) based on a previous pooled analysis of patients with known onset of ischemic stroke receiving thrombolysis. [4]

As the patients included in this meta-analysis had mild to moderate strokes (median NIHSS score of 7), results may not be applicable to patients with severe stroke and large core. A dose-effect of alteplase (0.6 mg/kg vs. 0.9 mg/kg) could not be definitively concluded, as only one of the four trials used the lower dose. Despite the fact that 60% of the patients were from the WAKE-UP trial, it’s concluded that in stroke patients with unknown time of onset with a DWI-FLAIR or perfusion mismatch, IV alteplase resulted in better functional outcomes at 90 days than placebo or standard care. A net benefit was observed for all functional outcomes regardless of the increased risk of sICH. [4]

A 2025 meta-analysis included 6 randomized controlled trials (N= 1,955 patients) to assess use of tenecteplase in AIS patients presenting after 4.5 hours of symptom onset or wake up AIS. A pooled analysis found treatment with tenecteplase was associated with a significantly improved excellent functional outcome on 90 days (OR 1.35, 95% CI 1.12 to 1.64) compared to control. However, no significant association was found between tenecteplase and control for good functional outcome, all-cause death, sICH, or any ICH. Ultimately, administration of tenecteplase even in an extended time window for AIS patients resulted in more favorable neurological outcomes. [5]

References:

[1] Günkan A, Ferreira MY, Vilardo M, et al. Thrombolysis for Ischemic Stroke Beyond the 4.5-Hour Window: A Meta-Analysis of Randomized Clinical Trials. Stroke. 2025;56(3):580-590. doi:10.1161/STROKEAHA.124.048536
[2] Palaiodimou L, Katsanos AH, Turc G, et al. Tenecteplase for the treatment of acute ischemic stroke in the extended time window: a systematic review and meta-analysis. Ther Adv Neurol Disord. 2024;17:17562864231221324. Published 2024 Jan 6. doi:10.1177/17562864231221324
[3] Aladawi M, Abuawwad MT, Taha MJJ, et al. Tenecteplase Beyond 4.5 Hours in Acute Ischemic Stroke: A Systematic Review and Meta-Analysis of Randomized Clinical Trials. J Stroke. 2025;27(2):184-194. doi:10.5853/jos.2024.05715
[4] Thomalla G, Boutitie F, Ma H, et al. Intravenous alteplase for stroke with unknown time of onset guided by advanced imaging: systematic review and meta-analysis of individual patient data. Lancet. 2020;396(10262):1574-1584. doi:10.1016/S0140-6736(20)32163-2
[5] Ifzaal M, Bughio SA, Rizvi SAFA, et al. Efficacy and safety of tenecteplase administration in extended time window for acute ischemic stroke: An updated meta-analysis of randomized controlled trials. J Stroke Cerebrovasc Dis. 2025;34(7):108338. doi:10.1016/j.jstrokecerebrovasdis.2025.108338

Literature Review

A search of the published medical literature revealed 2 studies investigating the researchable question:

could you please provide a summary of the available literature on the use of TNK in extended window stroke? In particular, I would appreciate insights into efficacy vs. safety, as well as any available comparisons between TNK and alteplase in this context. Thank you!

Level of evidence

B - One high-quality study or multiple studies with limitations  Read more→



Please see Tables 1-2 for your response.


Tenecteplase for Stroke at 4.5 to 24 Hours with Perfusion-Imaging Selection
Design

Multicenter, double-blind, randomized, placebo-controlled trial

N= 458

Objective To test the hypothesis that intravenous tenecteplase, initiated 4.5 to 24 hours after stroke onset, would provide a benefit in patients with a large-vessel occlusion of the internal carotid artery or the first (M1) or second (M2) segments of the middle cerebral artery and had evidence of salvageable ischemic brain tissue identified on CT perfusion or MRI perfusion–diffusion studies
Study Groups

Tenecteplase (n= 228)

Placebo (n= 230)

Inclusion Criteria Patients at least 18 years of age with independent function before the stroke (baseline prestroke modified Rankin scale score, 0 to 2), who had an ischemic stroke and could receive tenecteplase or placebo 4.5 to 24 hours after the time they were last known to be well, with a NIHSS score of at least 5, attributed to occlusion of the internal carotid artery or the M1 or M2 segment of the middle cerebral artery, and evidence of salvageable brain tissue
Exclusion Criteria Not specified in the provided text
Methods Patients were randomly assigned to receive either tenecteplase (0.25 mg per kilogram of body weight, up to 25 mg) or placebo, administered as an intravenous bolus over 5 seconds. Randomization was stratified by age, occlusion site, baseline NIHSS score, and center capability for endovascular treatment. Endovascular thrombectomy was performed according to standard care at each site
Duration March 2019 through December 2022
Outcome Measures

Primary: Ordinal score on the modified Rankin scale at day 90

Secondary: Functional independence at day 90, recanalization at 24 hours, angiographic reperfusion at the completion of endovascular thrombectomy, median NIHSS score at day 90, Barthel Index score at day 90, Glasgow Outcome Scale at day 90

Baseline Characteristics   Tenecteplase (N= 228) Placebo (N= 230)
Median age (IQR) — yr 72 (62–79) 73 (63–82)
Female sex  122 (53.5%) 123 (53.5%)
Race or ethnic group — no. (%) - White 169 (74.1%) 170 (73.9%)
Median NIHSS score (IQR) 12 (8–17) 12 (8–18)
Occlusion site- M1 segment 110 (48.2%) 117 (50.9%)
Endovascular thrombectomy performed  176 (77.2%) 178 (77.4%)
Results   Tenecteplase (N= 228) Placebo (N= 230) Adjusted Odds Ratio (95% CI)
Median score on the modified Rankin scale at 90 days (IQR) 3 (1–5) 3 (1–4) 1.13 (0.82–1.57)
Functional independence at 90 days  104/226 (46.0%) 97/229 (42.4%) 1.18 (0.80–1.74)
Recanalization at 24 hr  148/193 (76.7%) 124/194 (63.9%) 1.89 (1.21–2.95)
Reperfusion at the conclusion of endovascular thrombectomy  156/175 (89.1%) 152/178 (85.4%) 1.42 (0.75–2.67)
Adverse Events Mortality at 90 days was 19.7% in the tenecteplase group and 18.2% in the placebo group. The incidence of symptomatic intracranial hemorrhage was 3.2% in the tenecteplase group and 2.3% in the placebo group.
Study Author Conclusions Tenecteplase therapy initiated 4.5 to 24 hours after stroke onset in patients with occlusions of the middle cerebral artery or internal carotid artery did not result in better clinical outcomes than placebo. The incidence of symptomatic intracerebral hemorrhage was similar in both groups.
Critique The study was well-designed as a multicenter, double-blind, randomized, placebo-controlled trial, which strengthens the validity of the findings. However, the lack of significant difference in outcomes between the tenecteplase and placebo groups may limit the clinical impact of the findings. The study's reliance on imaging criteria for patient selection is a strength, but the high proportion of patients undergoing endovascular thrombectomy may have influenced the results, potentially masking any benefit of tenecteplase alone. Additionally, the study did not explore the effects of tenecteplase in patients who did not undergo thrombectomy, which could have provided further insights into its efficacy in different clinical scenarios.
References:

Albers GW, Jumaa M, Purdon B, et al. Tenecteplase for Stroke at 4.5 to 24 Hours with Perfusion-Imaging Selection. N Engl J Med. 2024;390(8):701-711. doi:10.1056/NEJMoa2310392

Thrombolysis With Alteplase at 0.6 mg/kg for Stroke With Unknown Time of Onset: A Randomized Controlled Trial
Design

Investigator-initiated, multicenter, randomized, open-label, blinded-end point trial

N= 131

Objective To assess whether lower-dose alteplase at 0.6 mg/kg is efficacious and safe for acute fluid-attenuated inversion recovery-negative stroke with unknown time of onset
Study Groups

Alteplase group (n= 70)

Control group (n= 61)

Inclusion Criteria Patients with stroke symptoms on awaking or with unknown time of onset, presented >4.5 hours since last-known-well and within 4.5 hours after symptom recognition, age ≥20 years, premorbid modified Rankin Scale (mRS)
Exclusion Criteria Mild stroke with NIHSS <5 or severe stroke with NIHSS >25, contraindications for MRI, planned surgery or endovascular reperfusion, pregnant or lactating, life expectancy of 6 months or less, intracranial hemorrhage or large infarct with ASPECTS ≤4
Methods Patients were randomized to receive either intravenous alteplase at 0.6 mg/kg or standard treatment. Alteplase was administered with 10% bolus and 90% by 60-minute infusion. MRI was used to confirm negative FLAIR pattern. Antithrombotics were prohibited in the alteplase group within the initial 25 hours
Duration May 1, 2014 to July 10, 2018
Outcome Measures

Primary: Favorable outcome (90-day modified Rankin Scale score of 0–1)

Secondary: mRS score 0–2 at 90 days, category shift in NIHSS score at 24 hours and 7 days, recanalization of the culprit artery, infarct volume and growth on FLAIR

Baseline Characteristics   Alteplase Group (n=70) Control Group (n=61)
Age, y; mean±SD 73.2±12.4 75.8±11.9
Female  25 (36%) 30 (49%)
Hypertension 49 (70%) 41 (67%)
Diabetes mellitus 14 (20%) 12 (20%)
Dyslipidemia 23 (33%) 23 (38%)
Atrial fibrillation 27 (39%) 21 (34%)
History of ischemic stroke/TIA 8 (11%) 14 (23%)
NIHSS score, median (IQR) 7 (4–13) 7 (5–12)
Results   Alteplase Group (n=70) Control Group (n=61) Effect Variable p-value
Favorable outcome at 90 d 32/68 (47.1%) 28/58 (48.3%) Relative risk 0.97 (0.68–1.41) 0.89
mRS score 0–2 at 90 d 40/68 (58.8%) 35/58 (60.3%) Relative risk 0.97 (0.73–1.30) 0.86
Recanalization of culprit artery on MRA at 22–36 h 14/19 (73.7%) 9/22 (40.9%) Relative risk 1.80 (1.02–3.64) 0.04
Adverse Events Symptomatic intracranial hemorrhage occurred in 1.4% of the alteplase group and 0% of the control group. No major extracranial bleeding was reported in either group. Death at 90 days was 2.8% in the alteplase group and 3.3% in the control group.
Study Author Conclusions No difference in favorable outcome was seen between alteplase and control groups among patients with ischemic stroke with unknown time of onset. The safety of alteplase at 0.6 mg/kg was comparable to that of standard treatment. Early study termination precludes any definitive conclusions.
Critique The study was limited by its premature termination, resulting in a smaller sample size than planned, which precludes definitive conclusions. The open treatment design may have influenced treatment processes. The exclusion of patients eligible for mechanical thrombectomy may have introduced selection bias. The use of a lower dose of alteplase compared to other trials may affect comparability of results.
References:

Koga M, Yamamoto H, Inoue M, et al. Thrombolysis With Alteplase at 0.6 mg/kg for Stroke With Unknown Time of Onset: A Randomized Controlled Trial. Stroke. 2020;51(5):1530-1538. doi:10.1161/STROKEAHA.119.028127