The New England Journal of Medicine JULY 15, 2020

Ticagrelor and Aspirin or Aspirin Alone in Acute Ischemic Stroke or TIA

S. Claiborne Johnston, Pierre Amarenco, Hans Denison, Scott R. Evans, et al.

Bottom Line

In patients with mild-to-moderate acute non-cardioembolic ischemic stroke or high-risk transient ischemic attack, a 30-day regimen of ticagrelor combined with aspirin reduced the risk of the composite of stroke or death compared to aspirin alone, albeit with an increased risk of severe bleeding.

Key Findings

1. The primary outcome of stroke or death within 30 days occurred in 5.5% of patients in the ticagrelor-aspirin group compared to 6.6% in the aspirin-alone group (Hazard Ratio [HR] 0.83; 95% Confidence Interval [CI] 0.71-0.96; P=0.02).
2. Severe bleeding, defined by the GUSTO criteria, was significantly more frequent in the ticagrelor-aspirin group (0.5%) compared to the aspirin-alone group (0.1%) (Hazard Ratio 3.99; 95% CI 1.74-9.14; P=0.001).
3. Intracranial hemorrhage occurred in 0.4% of patients in the ticagrelor-aspirin group versus 0.1% in the aspirin-alone group (P=0.005).
4. There was no statistically significant difference in the incidence of disability between the two study arms at 30 days.

Study Design

Design
RCT
Double-Blind
Sample
11,016
Patients
Duration
30 days
Median
Setting
Multicenter, international
Population Patients aged 40 years or older with mild-to-moderate acute non-cardioembolic ischemic stroke (NIHSS score ≤5) or high-risk TIA (ABCD2 score ≥6 or symptomatic arterial stenosis) within 24 hours of symptom onset.
Intervention Ticagrelor (180 mg loading dose, followed by 90 mg twice daily) plus aspirin for 30 days.
Comparator Matching placebo plus aspirin (loading dose 300-325 mg, followed by 75-100 mg daily) for 30 days.
Outcome Composite of stroke or death within 30 days.

Study Limitations

The study was limited to a 30-day follow-up period, leaving the risk-benefit profile beyond this short-term window unclear.
The trial excluded patients who received intravenous or mechanical reperfusion therapies, potentially limiting the generalizability of results to those receiving standard acute interventions.
The increased incidence of severe and intracranial bleeding, while anticipated with dual antiplatelet therapy, necessitates careful patient selection.

Clinical Significance

The THALES trial provides evidence for a pharmacological strategy to reduce early recurrent stroke in high-risk patients. While dual antiplatelet therapy with ticagrelor and aspirin offers a reduction in ischemic events, the increased risk of severe bleeding requires clinicians to weigh the potential ischemic benefit against the heightened risk of intracranial hemorrhage on an individual patient basis.

Historical Context

Following the success of the CHANCE and POINT trials, which demonstrated that dual antiplatelet therapy (aspirin and clopidogrel) reduced early recurrence of ischemic events in minor stroke or high-risk TIA, THALES evaluated whether an alternative P2Y12 inhibitor, ticagrelor—which acts directly and is not dependent on cytochrome P450 activation—could provide similar or superior protection.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

Ticagrelor and aspirin work through different pathways to inhibit platelet aggregation. What are their specific molecular targets, and why is dual antiplatelet therapy (DAPT) theoretically superior to monotherapy in the hyperacute phase of ischemic stroke?

Key Response

Aspirin irreversibly inhibits cyclooxygenase-1 (COX-1), preventing the synthesis of thromboxane A2, while ticagrelor is a direct-acting, reversible P2Y12 receptor antagonist that inhibits ADP-induced platelet activation. Using both targets complementary pathways of platelet activation, which is crucial in the first 24-72 hours after a stroke when the plaque is most unstable and platelet reactivity is at its peak.

Resident
Resident

The THALES trial utilized a 30-day regimen of ticagrelor and aspirin. How does this duration and the inclusion criteria (NIHSS ≤ 5) compare to the POINT and CHANCE trials, and how does this affect your choice of DAPT for a patient with a high-risk TIA?

Key Response

CHANCE and POINT used clopidogrel-aspirin. CHANCE (China) treated for 21 days, while POINT (International) treated for 90 days. THALES showed that 30 days of ticagrelor-aspirin reduces stroke risk but increases severe bleeding (GUSTO criteria). Residents must balance the higher potency of ticagrelor against the increased bleeding risk compared to clopidogrel, especially in patients who may be clopidogrel 'non-responders' due to CYP2C19 polymorphisms.

Fellow
Fellow

In the THALES trial, the benefit of ticagrelor-aspirin was consistent across subgroups, but the risk of intracranial hemorrhage was notably higher in the DAPT group. How should the presence of underlying cerebral small vessel disease (e.g., high burden of microbleeds) influence your interpretation of the 'net clinical benefit' for a specific patient?

Key Response

While the primary composite outcome favored DAPT, the hazard ratio for severe bleeding was 3.99. Fellows must integrate neuroimaging markers: patients with high microbleed burdens or those on concurrent anticoagulation (excluded from THALES) are at significantly higher risk for hemorrhagic transformation or ICH, potentially negating the 1.1% absolute risk reduction in ischemic stroke seen in the trial.

Attending
Attending

The NNT to prevent one primary outcome event in THALES was 92, while the NNH for a severe bleeding event was 263. Given these numbers, how do you incorporate the THALES data into your institutional protocols for patients who present at the 12-hour mark, outside the traditional window for some previous DAPT protocols?

Key Response

THALES allowed enrollment up to 24 hours after symptom onset, broader than the 12-hour window in CHANCE. This provides a practice-changing evidence base for late-presenting minor strokes. Attendings should emphasize that while the NNT is favorable, the 'real-world' NNH may be lower in elderly populations with higher frailty than the trial participants, necessitating a personalized approach to the 30-day duration.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

Critique the use of the GUSTO bleeding criteria in THALES versus the BARC or TIMI scales. How might the choice of bleeding definition mask the 'nuisance' bleeding that leads to premature treatment discontinuation in a 30-day trial of a P2Y12 inhibitor?

Key Response

GUSTO criteria focus on 'severe' or 'life-threatening' events, which are rare but catastrophic. However, ticagrelor is known to cause higher rates of dyspnea and minor/moderate bleeding (which would be captured by BARC). If 'nuisance' bleeding leads to early cessation of therapy in the DAPT arm, the intention-to-treat analysis might underestimate both the true preventive efficacy and the true total bleeding burden of the drug.

Journal Editor
Journal Editor

The THALES trial reported a statistically significant reduction in stroke or death, but no significant difference in the secondary outcome of disability (mRS > 2). As a reviewer, how would you evaluate the clinical significance of preventing a 'minor recurrent stroke' if it does not result in a measurable shift in global functional outcomes at 30 days?

Key Response

Editors look for 'patient-important outcomes.' If the reduction in strokes (mostly minor) doesn't translate to better functional independence (mRS), the value proposition of a more expensive and higher-risk drug like ticagrelor is challenged. A tough reviewer would flag the discrepancy between the primary endpoint (event-based) and the functional endpoint (disability-based) as a limitation in the trial's impact on long-term quality of life.

Guideline Committee
Guideline Committee

Current AHA/ASA guidelines (2021) give a Class 2a recommendation for ticagrelor plus aspirin in minor stroke/high-risk TIA. What specific evidence from THALES justifies its place alongside the Class 1a recommendation for clopidogrel plus aspirin, and should the guidelines specify its use only in the absence of clopidogrel-related genetic or drug-drug interactions?

Key Response

The 1a recommendation for clopidogrel is based on multiple large trials (CHANCE/POINT). THALES provides a high-quality alternative (Level B-R), especially useful for the ~30% of the population with CYP2C19 loss-of-function alleles who do not metabolize clopidogrel effectively. The committee must decide if the increased bleeding risk of ticagrelor justifies it as a universal first-line option or if it should remain a secondary choice behind clopidogrel for most patients.

Clinical Landscape

Noteworthy Related Trials

2013

CHANCE Trial

n = 5,170 · NEJM

Tested

Clopidogrel plus aspirin

Population

Patients with high-risk TIA or minor ischemic stroke

Comparator

Aspirin alone

Endpoint

Stroke (ischemic or hemorrhagic) within 90 days

Key result: Dual antiplatelet therapy with clopidogrel and aspirin significantly reduced the risk of stroke compared to aspirin alone.
2016

SOCRATES Trial

n = 13,199 · NEJM

Tested

Ticagrelor monotherapy

Population

Patients with acute ischemic stroke or high-risk TIA

Comparator

Aspirin monotherapy

Endpoint

Time to first event of stroke, myocardial infarction, or death at 90 days

Key result: Ticagrelor was not superior to aspirin in reducing the rate of the primary composite endpoint in patients with acute ischemic stroke or TIA.
2018

POINT Trial

n = 4,881 · NEJM

Tested

Clopidogrel plus aspirin

Population

Patients with minor ischemic stroke or high-risk TIA

Comparator

Aspirin alone

Endpoint

Composite of ischemic stroke, myocardial infarction, or death from vascular causes at 90 days

Key result: Dual antiplatelet therapy reduced major ischemic events but was associated with an increase in major hemorrhage compared to aspirin alone.

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