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Neurology Clinical Trial Database

ARAIS

Argatroban Plus Recombinant Tissue-Type Plasminogen Activator for Acute Ischemic Stroke

Year of Publication: 2023

Authors: Hui-Sheng Chen, Yu Cui, Zhong-He Zhou, ..., for the ARAIS Investigators

Journal: JAMA

Citation: JAMA. 2023;329(8):640-650. doi:10.1001/jama.2023.0550

Link: https://jamanetwork.com/journals/jama/fullarticle/2801517


Clinical Question

Does adding argatroban to intravenous alteplase improve neurologic function in patients with acute ischemic stroke compared to alteplase alone?

Bottom Line

Among patients with acute ischemic stroke, treatment with argatroban plus intravenous alteplase compared with alteplase alone did not result in a significantly greater likelihood of excellent functional outcome at 90 days.

Major Points

  • Multicenter, open-label, blinded endpoint RCT conducted at 50 hospitals in China
  • 817 patients randomized with median age 65 years, median NIHSS score 9, 29.1% women
  • 93% trial completion rate (760/817 patients)
  • Primary outcome (mRS 0-1 at 90 days) occurred in 63.8% with argatroban plus alteplase vs 64.9% with alteplase alone (RD -1.0%, 95% CI -8.1% to 6.1%, P=0.78)
  • No significant differences in any secondary outcomes including favorable outcome, early neurologic improvement/deterioration, or vascular events
  • Similar safety profile between groups with symptomatic ICH rates of 2.1% vs 1.8%
  • Lower proportion of patients with large artery occlusion (20.8%) compared to previous studies may explain neutral results
  • Only 23.2% of patients met target APTT at 2 hours, taking approximately 5 hours to reach target
  • No evidence of effect modification across prespecified subgroups

Design

Study Type: Multicenter, open-label, blinded endpoint randomized clinical trial

Randomization: 1

Blinding: Open-label for treatment assignment; blinded endpoint assessment for primary outcome (mRS) and adverse events by assessors unaware of treatment assignment

Enrollment Period: January 18, 2019 to October 30, 2021

Follow-up Duration: 90 days

Centers: 50

Countries: China

Sample Size: 817

Analysis: Full analysis set (all randomized participants with at least 1 post-baseline efficacy evaluation). Generalized linear models (GLMs) for primary and secondary outcomes. Cox regression for time-to-event outcomes. Sensitivity analyses included last observation carried forward, worst-case scenario, best-case scenario, and multiple imputation. SPSS version 23 and R version 4.1.0 used for statistical analyses.


Inclusion Criteria

  • Adults aged 18 to 80 years
  • Acute ischemic stroke at time of randomization
  • Baseline NIHSS score >6
  • Enrolled up to 4.5 hours after symptom onset (time last seen well)
  • CT or MRI confirmation of ischemic stroke

Exclusion Criteria

  • Disability in the community (mRS score ≥2) before stroke
  • History of intracerebral hemorrhage
  • Gastrointestinal or urinary tract bleeding in last 30 days
  • Need for concomitant use of anticoagulants other than argatroban

Arms

FieldArgatroban plus alteplaseControl
InterventionIntravenous alteplase (0.9 mg/kg; maximum dose 90 mg; 10% as 1-minute bolus, remaining over 1 hour) within 4.5 hours of symptom onset, plus argatroban (100 μg/kg IV bolus over 3-5 minutes within 1 hour of alteplase bolus, followed by 1.0 μg/kg/min infusion for 48 hours). Argatroban infusion adjusted to target APTT of 1.75× baseline (±10%). Standard guideline-based treatments also provided.Intravenous alteplase (0.9 mg/kg; maximum dose 90 mg; 10% as 1-minute bolus, remaining over 1 hour) within 4.5 hours of symptom onset. Standard guideline-based treatments also provided.
Duration48 hours of argatroban infusionSingle alteplase administration

Outcomes

OutcomeTypeControlInterventionHR / OR / RRP-value
Excellent functional outcome, defined as modified Rankin Scale score of 0 to 1 at 90 daysPrimary238/367 (64.9%)210/329 (63.8%)1.02%0.78
Favorable functional outcome (mRS 0-2) at 90 daysSecondary280/367 (76.3%)250/329 (76.0%)RR 1.00 (0.92-1.08)0.93
Early neurologic improvement within 48 hours (≥2-point decrease in NIHSS)Secondary269 (67.9%)251 (69.0%)RR 1.03 (0.84-1.27)0.76
Early neurologic deterioration within 48 hours (≥4-point increase in NIHSS)Secondary20 (5.1%)13 (3.6%)RR 0.71 (0.36-1.40)0.31
Change in NIHSS score at day 14 from baselineSecondaryMedian -0.81Median -0.85GMR -0.03 (-0.16 to 0.11)0.69
Stroke or other vascular events within 90 daysSecondary1/367 (0.3%)1/329 (0.3%)HR 1.12 (0.07-17.94)0.94
mRS score distribution at 90 days (ordinal analysis)SecondaryOR 1.06 (0.81-1.39)0.66
Symptomatic intracranial hemorrhage (ECASS-II)Adverse7 (1.8%)8 (2.1%)
Symptomatic intracranial hemorrhage (SITS-MOST)Adverse7 (1.8%)8 (2.1%)
Symptomatic intracranial hemorrhage (NINDS)Adverse14 (3.5%)13 (3.4%)
Parenchymal hematoma type 2Adverse10 (2.5%)9 (2.3%)
Parenchymal hematoma type 1Adverse8 (2.0%)7 (1.8%)
Major systemic bleedingAdverse2 (0.5%)1 (0.3%)
Other bleeding eventsAdverse10 (2.5%)14 (3.7%)
Other most common adverse eventsAdverse43 (10.8%)46 (12.0%)

Subgroup Analysis

Prespecified subgroup analysis showed no evidence of effect modification for the primary outcome by age (<65 vs ≥65 years), sex, NIHSS score at randomization (6-9 vs >9), endovascular therapy (yes vs no), large artery occlusion (yes vs no), time from symptom onset to thrombolysis (<3 hours vs 3-4.5 hours), premorbid function (mRS score), or history of stroke/TIA. All interaction P-values were non-significant.


Criticisms

  • Sample size not fully achieved in argatroban group (n=364 vs planned n=367) due to higher dropout rate, potentially reducing statistical power
  • Open-label design could introduce bias, though primary endpoint was assessed by blinded evaluators
  • Lower proportion of patients with large artery occlusion (20.8%) compared to previous positive studies (51.1%-100%), which may explain negative results
  • Only 23.2% of patients met target APTT at 2 hours; took approximately 5 hours to reach target APTT, suggesting suboptimal argatroban dosing
  • Large difference between assumed (21%) and observed (64%) rates of excellent functional outcome suggests power calculation may have been based on overly pessimistic assumptions
  • Endovascular thrombectomy used infrequently, limiting generalizability to comprehensive stroke centers
  • Conducted only in Chinese population, limiting generalizability to other ethnic groups
  • Higher dropout rate in argatroban group (21 withdrew consent) vs control group (12 withdrew consent) may introduce attrition bias
  • Per-protocol analysis excluded many patients (39 from argatroban group, 29 from alteplase group) due to protocol deviations
  • Low-dose argatroban regimen (1 μg/kg/min) compared to high-dose studies (3 μg/kg/min) may have limited efficacy

Funding

National Natural Science Foundation of China (81825007, 8207147), Beijing Outstanding Young Scientist Program (BJJWZYJH01201910025030), National Key R&D Program of China (2017YFC1308200), Science and Technology Project Plan of Liao Ning Province (2019JH2/10300027)

Based on: ARAIS (JAMA, 2023)

Authors: Hui-Sheng Chen, Yu Cui, Zhong-He Zhou, ..., for the ARAIS Investigators

Citation: JAMA. 2023;329(8):640-650. doi:10.1001/jama.2023.0550

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