The New England Journal of Medicine AUGUST 30, 2012

Secondary Prevention of Small Subcortical Strokes (SPS3) Trial

Oscar R. Benavente, Robert G. Hart, et al. (for the SPS3 Investigators)

Bottom Line

The SPS3 trial investigated antiplatelet therapy and blood pressure management in patients with recent lacunar stroke, finding that dual antiplatelet therapy (aspirin plus clopidogrel) increased bleeding risks without reducing stroke recurrence compared to aspirin alone, while intensive blood pressure control (<130 mmHg) showed a trend toward benefit in reducing stroke risk.

Key Findings

1. Dual antiplatelet therapy (aspirin 325 mg/day + clopidogrel 75 mg/day) did not significantly reduce the primary endpoint of recurrent stroke compared to aspirin monotherapy (hazard ratio 0.92; 95% CI 0.72-1.16; p=0.45).
2. Dual antiplatelet therapy was associated with a significant increase in major hemorrhage (2.1% per year vs 1.1% per year; hazard ratio 1.97; 95% CI 1.41-2.71; p<0.001) and all-cause mortality (2.2% per year vs 1.4% per year; hazard ratio 1.52; 95% CI 1.14-2.04; p=0.004).
3. Intensive systolic blood pressure control (<130 mmHg) compared to a target of 130-149 mmHg resulted in a non-significant reduction in all strokes (hazard ratio 0.81; 95% CI 0.64-1.03; p=0.08), though it demonstrated a significant reduction in the secondary endpoint of intracranial hemorrhage.
4. The antiplatelet arm of the trial was terminated early by the Data and Safety Monitoring Board due to futility and increased adverse event rates.

Study Design

Design
RCT
Double-Blind (Antiplatelet arm), Open-Label (BP arm)
Sample
3,020
Patients
Duration
3.7 yr
Median
Setting
Multicenter, international
Population Patients with a recent (within 180 days) symptomatic small subcortical (lacunar) stroke confirmed by MRI.
Intervention Combination antiplatelet therapy (aspirin 325 mg + clopidogrel 75 mg) and intensive SBP target (<130 mmHg).
Comparator Aspirin (325 mg) + placebo and usual SBP target (130-149 mmHg).
Outcome Time to recurrence of any stroke (ischemic or hemorrhagic).

Study Limitations

The antiplatelet arm was terminated prematurely, potentially limiting the statistical power to detect smaller, but clinically relevant, differences in outcomes.
The median time to enrollment was 62 days after the index stroke, a period where the risk of recurrent stroke is significantly lower compared to the acute phase, potentially attenuating the observed efficacy of antiplatelet therapies.
The trial utilized a high dose of aspirin (325 mg), which may have contributed to the increased bleeding risk observed in the combination therapy arm.
The blood pressure arm was open-label, introducing potential for bias in the management and reporting of endpoints.

Clinical Significance

The findings discourage the routine use of dual antiplatelet therapy (aspirin plus clopidogrel) for the secondary prevention of lacunar stroke, as the bleeding risk outweighs any potential benefit. The study supports a systolic blood pressure target of <130 mmHg as a safe and likely beneficial strategy for reducing stroke recurrence in patients with small vessel disease.

Historical Context

Prior to SPS3, the optimal secondary prevention strategies for lacunar stroke were unclear, and clinical practice varied. SPS3 was designed to address this knowledge gap by testing two therapeutic interventions in a large, systematic 2x2 factorial trial, setting a benchmark for evidence-based management of lacunar disease.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

What are the primary pathophysiological differences between the 'small subcortical' (lacunar) strokes studied in SPS3 and large-vessel atherosclerotic strokes, and how does this explain the differing responses to dual antiplatelet therapy?

Key Response

Lacunar strokes are primarily caused by small-vessel lipohyalinosis or microatheromas in the penetrating arteries, rather than the large-vessel plaque rupture and high-burden thrombus formation seen in carotid or middle cerebral artery disease. Because the underlying mechanism in small-vessel disease is often more related to chronic hypertensive damage than to acute arterial-arterial embolism, the added antithrombotic benefit of dual antiplatelet therapy (DAPT) is lower, while the bleeding risk—particularly into damaged microvessels—is significantly higher.

Resident
Resident

Given the results of the SPS3 trial, how should you tailor the secondary prevention strategy for a patient who presents with a pure motor stroke and a corresponding 10mm infarct in the internal capsule, specifically regarding the choice of antiplatelet and blood pressure target?

Key Response

SPS3 demonstrated that for lacunar strokes, aspirin monotherapy is safer and just as effective as the combination of aspirin and clopidogrel, which was associated with increased mortality and bleeding. Regarding blood pressure, the trial suggests targeting a systolic BP of <130 mmHg. While the primary endpoint for stroke reduction in the BP arm was not statistically significant (p=0.08), there was a significant 63% reduction in the risk of intracranial hemorrhage, justifying more intensive control than the standard <140 mmHg.

Fellow
Fellow

SPS3 results for intensive blood pressure control (target <130 mmHg) showed a non-significant reduction in all strokes but a significant reduction in intracranial hemorrhage. How should this be integrated with the results of the later SPRINT trial when managing patients with cerebral small vessel disease (CSVD) and multiple lacunar infarcts?

Key Response

Fellows must synthesize evidence across trials. While SPS3 trended toward benefit, SPRINT later confirmed that intensive BP control (<120 mmHg) reduced cardiovascular events and mortality in high-risk patients. In the context of CSVD, the reduction in ICH found in SPS3 is a critical safety signal. This suggests that for patients with lacunar strokes, an intensive BP target is likely beneficial not just for primary prevention of future ischemic events, but specifically for mitigating the high hemorrhagic risk associated with fragile, small-vessel arteriopathy.

Attending
Attending

In the era of the POINT and CHANCE trials, which advocate for short-term DAPT in minor stroke, how do you differentiate 'minor stroke' from 'lacunar stroke' in the acute setting to ensure you are not inadvertently applying SPS3-ineligible management to your patients?

Key Response

This is a critical teaching point. POINT and CHANCE focused on high-risk TIA or minor ischemic strokes (NIHSS ≤3) mostly of large-vessel or embolic origin, where short-term (21-90 days) DAPT is beneficial. SPS3 showed that long-term DAPT is harmful in lacunar strokes. In practice, unless the stroke is clearly lacunar on urgent MRI (small, deep lesion) and lacks large-vessel stenosis, clinicians often start short-term DAPT for 'minor strokes' as per POINT; however, if the mechanism is confirmed as purely small-vessel disease, SPS3 guides us to transition back to monotherapy to avoid long-term harm.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

SPS3 utilized a 2x2 factorial design. What are the statistical and power implications of using this design when one intervention (DAPT) is terminated early for harm, and how might the presence or absence of an 'interaction effect' between blood pressure and antiplatelet intensity complicate the interpretation of the blood pressure arm?

Key Response

Factorial designs assume no interaction between treatments. If DAPT increased bleeding (which it did), and lower BP targets reduce bleeding (which they did), the two interventions could have a sub-additive or masking effect on the primary endpoint of total stroke. The early termination of the antiplatelet arm reduces the total person-years of follow-up for the BP arm within a factorial framework, potentially contributing to the 'near-significant' p-value (0.08) by reducing the power to detect the intended effect size.

Journal Editor
Journal Editor

As a reviewer, what concerns would you raise regarding the 'open-label' nature of the blood pressure intervention in SPS3 compared to the double-blind antiplatelet arm, and how does the achieve-target vs. intention-to-treat analysis impact the study's validity?

Key Response

Reviewers would flag potential performance bias; clinicians knew which patients were in the 'intensive' group and may have provided better overall care (the 'Hawthorne effect'). Furthermore, achieving a target of <130 mmHg is difficult; if a significant portion of the 'standard' group also achieved low BP, the contrast between groups narrows, leading to a type II error. Editors look for whether the trialists accounted for these cross-overs and if the monitoring was rigorous enough to ensure the BP separation was maintained throughout the study.

Guideline Committee
Guideline Committee

The 2021 AHA/ASA secondary stroke prevention guidelines recommend a BP target of <130/80 mmHg (Class I, LOE B-R). Given that the SPS3 primary endpoint for BP was not statistically significant, what specific secondary outcomes or meta-analyses including SPS3 justify this high level of recommendation?

Key Response

Guideline committees look beyond the primary p-value. The 'Class I' recommendation is justified by the significant reduction in intracranial hemorrhage (a devastating subtype of stroke) seen in SPS3, combined with evidence from SPRINT and meta-analyses showing a linear relationship between lower BP and reduced stroke risk. Despite the 0.08 p-value for 'all stroke,' the totality of the evidence—specifically the ICH prevention in a population prone to microhemorrhages—is viewed as practice-defining.

Clinical Landscape

Noteworthy Related Trials

2001

PROGRESS Trial

n = 6,105 · Lancet

Tested

Perindopril-based blood pressure lowering

Population

Patients with a history of stroke or TIA

Comparator

Placebo

Endpoint

Total stroke

Key result: Active treatment with perindopril significantly reduced the risk of recurrent stroke by 28%.
2012

SPS3 Antiplatelet Arm

n = 3,020 · Lancet Neurol

Tested

Dual antiplatelet therapy (Clopidogrel + Aspirin)

Population

Patients with recent symptomatic lacunar stroke

Comparator

Aspirin alone

Endpoint

Recurrent stroke

Key result: Dual antiplatelet therapy did not reduce the rate of recurrent stroke but significantly increased the risk of major hemorrhage compared to aspirin alone.
2013

SPS3 Trial

n = 3,020 · NEJM

Tested

Intensive blood pressure control (<130 mm Hg)

Population

Patients with recent lacunar stroke

Comparator

Standard blood pressure control (130-149 mm Hg)

Endpoint

Recurrent stroke (ischemic or hemorrhagic)

Key result: Intensive blood pressure control did not significantly reduce the risk of recurrent stroke compared to standard control but showed a trend toward reduced intracranial hemorrhage.

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