The Lancet Neurology September 04, 2012

MRI profile and response to endovascular reperfusion after stroke (DEFUSE 2): a prospective cohort study

Maarten G Lansberg, Matus Straka, Stephanie Kemp, et al.

Bottom Line

The DEFUSE 2 prospective cohort study demonstrated that acute ischemic stroke patients with an MRI target mismatch profile who achieve endovascular reperfusion have significantly higher odds of a favorable clinical outcome compared to those without a target mismatch.

Key Findings

1. Among 138 enrolled patients, 99 could be assessed for both an initial MRI profile and reperfusion status after endovascular treatment.
2. Reperfusion was achieved in 59% (46 of 78) of patients with a target mismatch profile and 57% (12 of 21) of patients without target mismatch.
3. In the target mismatch group, early reperfusion was associated with a significantly higher likelihood of a favorable clinical response (adjusted OR 8.8, 95% CI 2.7-29.0).
4. In the no target mismatch group, reperfusion was not associated with a favorable clinical response (adjusted OR 0.2, 95% CI 0.0-1.6; p=0.003 for the difference between ORs).
5. Reperfusion was also associated with increased odds of a good functional outcome (modified Rankin Scale score 0-2) at 90 days in the target mismatch group (adjusted OR 4.0, 95% CI 1.3-12.2), but not in the no target mismatch group (OR 1.9, 95% CI 0.2-18.7).

Study Design

Design
Prospective Cohort Study
Single-Blind
Sample
138
Patients
Duration
90 days
Median
Setting
Multicenter, US and Europe
Population Consecutive adult patients with acute ischemic stroke scheduled to undergo endovascular treatment within 12 hours of symptom onset.
Intervention Early reperfusion (>50% reduction in perfusion-weighted lesion volume from baseline) following endovascular therapy.
Comparator No early reperfusion after endovascular therapy, stratified by the presence or absence of a baseline MRI target mismatch profile.
Outcome Favorable clinical response, defined as an improvement of ≥8 points on the National Institutes of Health Stroke Scale (NIHSS) between baseline and day 30, or an NIHSS score of 0-1 at day 30.

Study Limitations

Non-randomized, observational cohort design without a medical therapy control group.
Endovascular treatment decisions were made at the discretion of the treating physician, introducing potential selection bias.
The small sample size within the no target mismatch subgroup (n=21) limited statistical power for evaluating outcomes in that cohort.
Analysis of favorable clinical outcomes required adjusting for imbalances in baseline predictors, highlighting potential residual confounding by indication.

Clinical Significance

DEFUSE 2 provided foundational proof-of-concept evidence that automated MRI-based 'target mismatch' criteria (identifying a small ischemic core with a large volume of critically hypoperfused, salvageable tissue) can successfully select acute ischemic stroke patients who are highly likely to benefit from endovascular reperfusion. This validated the tissue-based paradigm for acute stroke triage and directly set the stage for later definitive, randomized trials like DEFUSE 3 and DAWN, which utilized similar imaging criteria to extend the therapeutic window for mechanical thrombectomy up to 24 hours.

Historical Context

Prior to DEFUSE 2, the efficacy of endovascular therapy for acute ischemic stroke, particularly beyond the traditional early time windows (e.g., 3-4.5 hours for IV tPA or 6-8 hours for early endovascular devices), was uncertain. The DEFUSE 1 trial had originally proposed the target mismatch concept in patients receiving intravenous alteplase. DEFUSE 2 applied automated RAPID perfusion imaging software to an endovascular cohort treated up to 12 hours from symptom onset. It verified that imaging-selected patients derive dramatic clinical benefit from recanalization, shifting the stroke field away from strict chronological time windows toward physiological, tissue-based windows.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

What is the physiological difference between the ischemic core and the ischemic penumbra, and how do diffusion-weighted imaging (DWI) and perfusion-weighted imaging (PWI) differentiate these two zones on an MRI?

Key Response

The ischemic core consists of irreversibly damaged tissue characterized by cytotoxic edema, which restricts water diffusion and appears bright on DWI. The penumbra is hypoperfused but viable tissue at risk of infarction, identified by delayed perfusion on PWI without restricted diffusion on DWI. The difference between the PWI lesion and DWI lesion defines the target mismatch.

Resident
Resident

How does the presence or absence of a target mismatch on MRI influence the decision-making process for pursuing endovascular therapy in acute ischemic stroke patients outside the standard early time window?

Key Response

Residents must understand that target mismatch helps identify patients who have salvageable brain tissue regardless of the exact time from symptom onset. Proceeding with EVT in patients without a mismatch carries a higher risk of reperfusion injury and hemorrhage without clinical benefit, whereas those with a mismatch are prime candidates for late-window EVT.

Fellow
Fellow

In the context of DEFUSE 2, how do specific volumetric thresholds (e.g., core volume less than 70 mL or mismatch ratio) define a target mismatch, and what are the limitations of applying these rigid thresholds in borderline clinical scenarios?

Key Response

Fellows should know that DEFUSE 2 defined target mismatch using specific criteria (e.g., mismatch ratio greater than or equal to 1.8, absolute mismatch volume greater than or equal to 15 mL, core less than 70 mL). Relying strictly on automated thresholds can be problematic in borderline cases due to imaging artifacts, the dynamic nature of stroke evolution, and individual variations in collateral circulation.

Attending
Attending

While DEFUSE 2 strongly supports EVT for target mismatch profiles, how should an attending balance advanced imaging findings with clinical gestalt, specifically in patients with severe baseline neurological deficits but equivocal mismatch profiles?

Key Response

Attendings must weigh imaging data against the patient's overall clinical picture, age, comorbidities, and baseline functional status. Over-reliance on imaging algorithms can lead to withholding potentially life-altering therapy or performing futile interventions. It highlights the art of medicine in integrating trials with personalized care.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

DEFUSE 2 was a prospective cohort study, not a randomized controlled trial. What are the inherent methodological limitations of using a cohort design to evaluate treatment response, and how might selection bias or confounding by indication have influenced the observed association between reperfusion and favorable outcomes?

Key Response

A cohort study cannot definitively prove causation because the decision to pursue endovascular therapy and the success of reperfusion are not randomized. Patients achieving reperfusion might have underlying biological differences (e.g., better collaterals, softer clots) that independently predict better outcomes, making confounding by indication a significant threat to internal validity.

Journal Editor
Journal Editor

As a reviewer, how would you critically evaluate the study's use of early-generation automated perfusion software (e.g., RAPID) for defining mismatch, and what concerns might arise regarding the reproducibility of these imaging parameters across different hospital settings and MRI vendors?

Key Response

Editors must scrutinize the generalizability and reproducibility of the imaging biomarker. Differences in MRI hardware, sequence parameters, and post-processing software versions can significantly alter calculated core and penumbra volumes. Ensuring that the study's automated thresholds are robust across heterogeneous real-world settings is a critical point for publication and external validity.

Guideline Committee
Guideline Committee

Based on the DEFUSE 2 findings and subsequent confirmatory randomized trials, how has the strength of recommendation for advanced imaging (CTP or MRI) changed in current AHA/ASA guidelines for selecting patients for endovascular thrombectomy in the extended 6-to-24 hour time window?

Key Response

DEFUSE 2 paved the way for phase 3 trials like DEFUSE 3 and DAWN. Current AHA/ASA guidelines give a Class I, Level of Evidence A recommendation for using CTP or DWI/PWI MRI to select patients for mechanical thrombectomy between 6 and 24 hours of last known normal. Guideline committees must utilize these foundational cohort data combined with later RCTs to establish and update the standard of care for late-window selection.

Clinical Landscape

Noteworthy Related Trials

2015

EXTEND-IA Trial

n = 70 · NEJM

Tested

Endovascular thrombectomy plus alteplase

Population

Ischemic stroke within 4.5 hours with large vessel occlusion and salvageable tissue on CT perfusion

Comparator

Alteplase alone

Endpoint

Reperfusion at 24 hours and early neurologic improvement

Key result: Endovascular therapy significantly improved reperfusion early neurologic recovery and functional outcome compared to alteplase alone.
2018

DEFUSE 3 Trial

n = 182 · NEJM

Tested

Endovascular thrombectomy plus standard medical therapy

Population

Ischemic stroke 6 to 16 hours from onset with salvageable tissue on perfusion imaging

Comparator

Standard medical therapy alone

Endpoint

Ordinal score on the modified Rankin scale at 90 days

Key result: Endovascular therapy resulted in a better functional outcome than medical therapy among patients treated 6 to 16 hours after stroke onset.
2018

DAWN Trial

n = 206 · NEJM

Tested

Endovascular thrombectomy plus standard medical therapy

Population

Ischemic stroke 6 to 24 hours from onset with clinical-core mismatch

Comparator

Standard medical therapy alone

Endpoint

Utility-weighted modified Rankin scale score at 90 days

Key result: Thrombectomy significantly improved clinical outcomes in patients with a mismatch between clinical deficit and infarct volume treated up to 24 hours.

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