The New England Journal of Medicine NOVEMBER 21, 2019

Ticagrelor with or without Aspirin in High-Risk Patients after Coronary Intervention (TWILIGHT)

Usman Baber, Ajay J. Kirtane, Dominick J. Angiolillo, et al.

Bottom Line

In high-risk patients undergoing percutaneous coronary intervention (PCI) who remained event-free after 3 months of dual antiplatelet therapy (DAPT), ticagrelor monotherapy significantly reduced the risk of clinically relevant bleeding compared to continued ticagrelor plus aspirin, without increasing the risk of ischemic events.

Key Findings

1. The primary endpoint of BARC type 2, 3, or 5 bleeding occurred in 4.0% of patients in the ticagrelor monotherapy group compared with 7.1% in the ticagrelor plus aspirin group (hazard ratio, 0.56; 95% CI, 0.45 to 0.68; P<0.001).
2. The risk of major ischemic events (composite of all-cause death, myocardial infarction, or stroke) was 3.9% in both the ticagrelor monotherapy group and the ticagrelor plus aspirin group (hazard ratio, 0.99; 95% CI, 0.78 to 1.25; P=0.95), meeting the criteria for noninferiority.
3. The benefit of reduced bleeding with ticagrelor monotherapy was observed across various high-risk subgroups, including those with complex PCI and those at high bleeding risk, with no significant increase in ischemic harm.

Study Design

Design
RCT
Double-Blind
Sample
7,119
Patients
Duration
12 mo
Median
Setting
Multicenter, 11 countries
Population Patients at high risk for ischemic or bleeding events who underwent successful PCI with at least one drug-eluting stent and remained event-free after 3 months of DAPT.
Intervention Ticagrelor monotherapy (90 mg twice daily) plus placebo
Comparator Ticagrelor (90 mg twice daily) plus aspirin (81-100 mg daily)
Outcome Bleeding Academic Research Consortium (BARC) type 2, 3, or 5 bleeding from randomization at 3 months to 15 months after the index procedure.

Study Limitations

The study design required patients to be event-free and adherent to DAPT for the initial 3 months, which may limit the generalizability of findings to patients who suffer early ischemic or bleeding events.
Patients with ST-segment elevation myocardial infarction (STEMI) or those requiring long-term oral anticoagulation were excluded, limiting the applicability of these findings to these specific populations.
As an event-driven trial, the results specifically reflect the risk-benefit balance in a high-risk population already tolerant of dual antiplatelet therapy.

Clinical Significance

The TWILIGHT trial provides robust evidence for an 'abbreviated DAPT' strategy, supporting the transition to P2Y12 inhibitor monotherapy (specifically ticagrelor) after a short (3-month) course of DAPT in selected high-risk patients post-PCI. This strategy optimizes the balance between bleeding reduction and ischemic protection, serving as a foundational study for shifting clinical practice toward de-escalation of antiplatelet therapy.

Historical Context

Historically, long-term DAPT (typically 12 months or longer) was the standard of care following PCI with drug-eluting stents to prevent stent thrombosis and recurrent ischemic events. However, the accumulation of evidence regarding the high burden of bleeding complications associated with prolonged DAPT in high-risk populations led to the exploration of shorter DAPT durations and monotherapy strategies, with TWILIGHT being a pivotal study to confirm safety and bleeding reduction benefits.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

How does the pharmacological mechanism of ticagrelor differ from that of aspirin, and why would removing aspirin after 3 months potentially reduce bleeding without losing anti-thrombotic protection in post-PCI patients?

Key Response

Aspirin irreversibly inhibits COX-1, leading to reduced thromboxane A2, which affects platelet activation but also impairs gastric mucosal protection. Ticagrelor is a reversible, direct-acting P2Y12 receptor antagonist that inhibits ADP-induced platelet aggregation. In the chronic phase after PCI, once the initial high-risk period for stent thrombosis has passed, the potent P2Y12 inhibition provided by ticagrelor is often sufficient to prevent ischemic events, while removing the COX-1 inhibition of aspirin reduces systemic bleeding risks, particularly gastrointestinal ones.

Resident
Resident

A patient with high-risk clinical features (e.g., diabetes and CKD) just reached the 3-month mark after a complex PCI and has had no ischemic or bleeding events. Based on TWILIGHT, what is the clinical rationale for switching to ticagrelor monotherapy instead of completing the traditional 12 months of DAPT?

Key Response

The TWILIGHT trial demonstrated that in patients who remain event-free for 3 months post-PCI, transitioning to ticagrelor monotherapy significantly reduced the risk of BARC 2, 3, or 5 bleeding (4.0% vs. 7.1%; HR 0.56) compared to continuing ticagrelor plus aspirin. Crucially, this was achieved without a significant increase in the composite ischemic endpoint of death, MI, or stroke, making it a safer strategy for high-risk patients who have successfully cleared the early high-thrombotic window.

Fellow
Fellow

The TWILIGHT trial enrolled 'high-risk' patients based on both clinical and angiographic criteria. How do these results compare with the GLOBAL LEADERS trial regarding the efficacy of ticagrelor monotherapy, and what role did the 3-month 'event-free' lead-in period play in the success of the TWILIGHT strategy?

Key Response

GLOBAL LEADERS failed to show superiority for its experimental 23-month ticagrelor monotherapy regimen (after 1 month of DAPT) compared to 12 months of standard DAPT. TWILIGHT succeeded likely because of its landmark design: it only randomized patients who were already event-free and adherent after 3 months of DAPT. This 'stable survivor' bias ensures that the monotherapy transition occurs after the highest risk period for early stent thrombosis has passed, refining the patient population to those most likely to benefit from a bleeding-reduction strategy.

Attending
Attending

Given the findings of TWILIGHT, should ticagrelor monotherapy after a short course of DAPT become the new 'default' for high-risk PCI patients, and how does this challenge the legacy role of aspirin as a life-long requirement for secondary prevention in coronary artery disease?

Key Response

TWILIGHT adds to a growing body of evidence (including TICO and SMART-CHOICE) suggesting that aspirin may be the 'weak link' in modern antiplatelet therapy—contributing significantly to bleeding without adding much to the potent P2Y12 inhibition offered by agents like ticagrelor. This represents a paradigm shift where P2Y12 monotherapy may eventually replace aspirin-based regimens, moving from 'dual therapy' to 'potent mono-therapy' as the standard for long-term management after the initial high-risk endothelialization phase of drug-eluting stents.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The TWILIGHT trial utilized a non-inferiority margin of 1.6% for the absolute difference in the composite ischemic endpoint. Critically evaluate the selection of this margin and discuss how the use of a 'per-protocol' vs. 'intention-to-treat' analysis in this context affects the robustness of the non-inferiority claim.

Key Response

Non-inferiority margins are often a point of contention; a 1.6% absolute margin is relatively wide given the low baseline event rate. In non-inferiority trials, an Intention-to-Treat (ITT) analysis can be anti-conservative because 'dilution' (patients not adhering to the protocol) tends to make the groups look more similar, biased toward the null (non-inferiority). A PhD-level critique would highlight that while TWILIGHT met its NI margin in both ITT and Per-Protocol sets, the wide margin and the landmark design (excluding early events) necessitate caution when generalizing to the highest-risk early-phase post-PCI populations.

Journal Editor
Journal Editor

As a reviewer, what are the primary threats to the external validity of the TWILIGHT trial results, specifically regarding the exclusion of patients with ST-segment elevation MI (STEMI) or those who experienced events during the 3-month DAPT run-in?

Key Response

The exclusion of STEMI patients and those who suffered ischemic or bleeding events in the first 90 days creates a 'highly selected' cohort of 'responders.' This significantly limits generalizability to the acute ACS setting where the risk of stent thrombosis is highest. A tough reviewer would flag that TWILIGHT is not a trial of 'ticagrelor monotherapy for PCI,' but rather a trial of 'switching to monotherapy in stable post-PCI survivors,' which is a critical distinction for clinical implementation.

Guideline Committee
Guideline Committee

Current ESC and ACC/AHA guidelines have historically recommended 6-12 months of DAPT post-PCI. How does the Level A evidence from TWILIGHT support a change in the Class of Recommendation for 'short-duration DAPT followed by P2Y12 monotherapy' in patients at high bleeding risk, and should this strategy be prioritized over clopidogrel-based de-escalation?

Key Response

TWILIGHT provides high-quality evidence to support a Class I or IIa recommendation for shortening DAPT to 3 months followed by ticagrelor monotherapy in high-risk patients. Compared to clopidogrel de-escalation (as seen in TOPIC or TROPICAL-ACS), the TWILIGHT strategy maintains a more potent antiplatelet effect while reducing bleeding by removing the second agent (aspirin) rather than switching to a weaker P2Y12 inhibitor. Guidelines like the 2023 ESC ACS update now reflect this, citing TWILIGHT as a basis for considering P2Y12 monotherapy as an alternative to standard DAPT durations.

Clinical Landscape

Noteworthy Related Trials

2018

GLOBAL LEADERS Trial

n = 15,968 · Lancet

Tested

Ticagrelor monotherapy after 1 month of DAPT

Population

Patients undergoing PCI with drug-eluting stents

Comparator

Standard DAPT (12 months of aspirin plus P2Y12 inhibitor)

Endpoint

All-cause mortality or new Q-wave MI at 2 years

Key result: Ticagrelor monotherapy after 1 month of DAPT did not significantly reduce the rate of death or new Q-wave MI compared with standard DAPT.
2019

STOPDAPT-2 Trial

n = 3,045 · JAMA

Tested

Clopidogrel monotherapy after 1 month of DAPT

Population

Patients undergoing PCI with drug-eluting stents

Comparator

Standard DAPT (12 months of aspirin plus clopidogrel)

Endpoint

Composite of cardiovascular death, MI, stroke, or bleeding at 12 months

Key result: Shortening DAPT to 1 month followed by clopidogrel monotherapy was superior to 12-month DAPT for bleeding and non-inferior for ischemic outcomes.
2019

Smart-CHOICE Trial

n = 2,993 · JAMA

Tested

P2Y12 inhibitor monotherapy after 3 months of DAPT

Population

Patients undergoing PCI

Comparator

Standard DAPT (12 months of aspirin plus P2Y12 inhibitor)

Endpoint

Major adverse cardiac and cerebrovascular events at 12 months

Key result: P2Y12 inhibitor monotherapy was non-inferior to continued DAPT for MACE, with a lower incidence of bleeding.

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