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ESPRIT

Aspirin plus dipyridamole versus aspirin alone after cerebral ischaemia of arterial origin (ESPRIT): randomised controlled trial

Year of Publication: 2006

Authors: Halkes PHA, van Gijn J, Kappelle LJ, ..., on behalf of the ESPRIT Study Group

Journal: Lancet

Citation: Lancet 2006;367(9523):1665-1673. DOI: 10.1016/S0140-6736(06)68734-5

Link: https://doi.org/10.1016/S0140-6736(06)68734-5

Bottom Line

Adding extended-release dipyridamole 200 mg twice daily to aspirin reduced the composite of vascular death, non-fatal stroke, non-fatal MI, or major bleeding by 20% (HR 0.80, 95% CI 0.66-0.98; ARR 1.0%/yr, NNT 104/yr) in patients with recent TIA or minor stroke of arterial origin; combined with ESPS-2 in an updated meta-analysis (RR 0.82, 95% CI 0.74-0.91) this established aspirin+dipyridamole as preferred antiplatelet therapy over aspirin monotherapy for secondary prevention after non-cardioembolic cerebral ischaemia — at the cost of a much higher discontinuation rate driven by dipyridamole-induced headache (34% vs 13%).

Major Points

  • Primary composite (vascular death, non-fatal stroke, non-fatal MI, or major bleeding) occurred in 173/1363 (13%) on aspirin+dipyridamole vs 216/1376 (16%) on aspirin alone; HR 0.80 (95% CI 0.66-0.98).
  • Absolute risk reduction 1.0% per year (95% CI 0.1-1.8), corresponding to NNT of 104 per year (95% CI 55-1006) to prevent one primary event.
  • Effect was consistent across prespecified subgroups (age, sex, randomisation scheme, ischaemic heart disease history, large vs small vessel disease, Asian vs non-Asian; smallest interaction p=0.18) and across post-hoc subgroups of aspirin dose (<40, 40-100, >100 mg) and time from event.
  • Updated meta-analysis pooling ESPRIT with 5 previous trials (ESPS-2, ACCSG-II, AICLA, Guiraud-Chaumeil, Kaye) yielded an overall RR of 0.82 (95% CI 0.74-0.91) for vascular death/stroke/MI across 7795 patients.
  • Major bleeding was numerically LOWER with combination therapy (35 vs 53 events; HR 0.67, 95% CI 0.44-1.03), a finding the authors attribute to chance; minor bleeding was similar (RR 1.03).
  • Tolerability was the main drawback: 470/1363 (34%) of combination patients discontinued vs 184/1376 (13%) on aspirin alone; dipyridamole-related headache was cited by 26% of those who stopped combination therapy.
  • Design features that broaden generalisability: open-label, pragmatic dosing (aspirin 30-325 mg allowed), 79 hospitals in 14 countries, minimal exclusion criteria, and blinded outcome adjudication by independent committee.
  • On-treatment analysis was similar to intention-to-treat (HR 0.82 vs 0.80), and observed event rate on aspirin (4.8%/yr) was lower than projected (6%/yr) but the trial's power was preserved by longer-than-planned 9722 patient-years of follow-up.

Design

Study Type: Randomized Controlled Trial (pragmatic, open-label with blinded outcome adjudication)

Randomization: 1

Blinding: Open-label treatment; blinded (PROBE-style) outcome adjudication by independent auditing committee

Enrollment Period: July 1, 1997 - December 31, 2005

Follow-up Duration: Mean 3.5 years (SD 2.0); total 9722 patient-years; close-out July 1 - December 31, 2005

Centers: 79

Countries: Netherlands, UK, Singapore, Italy, Belgium, Spain, Sweden, France, Germany, Austria, Portugal, Australia, Switzerland, USA

Sample Size: 2739

Analysis: Intention-to-treat (primary); on-treatment sensitivity analysis; Cox proportional-hazards for hazard ratios with 95% CI; five interim analyses per O'Brien-Fleming; updated meta-analysis with prior trials; 24 patients from one hospital excluded for incomplete data


Inclusion Criteria

  • Referred to a participating hospital within 6 months of a qualifying event
  • Qualifying event: transient ischaemic attack (including transient monocular blindness) OR minor ischaemic stroke
  • Modified Rankin scale ≤3 at randomisation (some restrictions but still independent or better)
  • Presumed arterial (non-cardioembolic) origin of cerebral ischaemia
  • Written informed consent

Exclusion Criteria

  • Possible cardiac source of embolism (atrial fibrillation on ECG, valvular heart disease, or recent myocardial infarction)
  • Cerebral ischaemia associated with high-grade carotid stenosis for which carotid endarterectomy or endovascular treatment was planned
  • Any blood coagulation disorder
  • Any contraindication to aspirin or to dipyridamole
  • Limited life expectancy

Baseline Characteristics

CharacteristicAspirin + Dipyridamole (n=1363)Aspirin alone (n=1376)
Mean Age (years)63 (SD 11)63 (SD 11)
Age ≥75 years~16% overall (n=450 in both arms combined)
Male897 (66%)892 (65%)
Qualifying event - Transient monocular blindness62 (5%)79 (6%)
Qualifying event - Transient ischaemic attack403 (30%)376 (27%)
Qualifying event - Minor ischaemic stroke895 (66%)921 (67%)
Time from event to randomisation <1 week149 (11%)151 (11%)
Time from event to randomisation 1 week - 1 month303 (23%)274 (20%)
Time from event to randomisation 1-6 months890 (66%)931 (69%)
Rankin 0 (no symptoms)588 (43%)574 (42%)
Rankin 1 (minor symptoms, no limitations)450 (33%)468 (34%)
Rankin 2 (some restrictions, no help)243 (18%)249 (18%)
Rankin 3 (help needed, still independent)77 (6%)84 (6%)
Previous stroke159 (12%)155 (11%)
Angina pectoris132 (10%)130 (9%)
Previous myocardial infarction91 (7%)93 (7%)
Intermittent claudication75 (6%)53 (4%)
Prior vascular intervention80 (6%)79 (6%)
Diabetes mellitus260 (19%)252 (18%)
Hypertension814 (60%)817 (59%)
Hyperlipidaemia634 (47%)638 (46%)
Current cigarette smoking484 (36%)512 (37%)
Systolic BP (mm Hg, mean, SD)152 (24)152 (23)
Diastolic BP (mm Hg, mean, SD)86 (12)86 (12)
Large vessel disease405 (30%)430 (31%)
Small vessel disease687 (50%)690 (50%)
Unspecified vessel type271 (20%)256 (19%)
Aspirin at time of qualifying event319 (23%)309 (22%)
Aspirin dose 30 mg576 (42%)635 (46%)
Aspirin dose 75 mg209 (15%)206 (15%)
Aspirin dose 100 mg316 (23%)340 (25%)
Median aspirin dose (mg)75 (range 30-325)75 (range 30-325)
Used extended-release dipyridamole1131 (83%)

Arms

FieldAspirin + DipyridamoleControl
InterventionAspirin 30-325 mg once daily (median 75 mg) PLUS dipyridamole 200 mg twice daily (extended-release in 83%); either as fixed-dose combination or free combinationAspirin 30-325 mg once daily (median 75 mg), dose left to local physician discretion
DurationMean 3.5 years (up to 8 years enrolment; centres could close follow-up after 5 years)Mean 3.5 years

Outcomes

OutcomeTypeControlInterventionHR / OR / RRP-value
Composite of death from all vascular causes, non-fatal stroke, non-fatal myocardial infarction, or non-fatal major bleeding complication — whichever event occurred first (intention-to-treat)Primary216/1376 (16%)173/1363 (13%)0.8<0.05 (CI excludes 1)
Death from all causesSecondary107 (7.8%)93 (6.8%)0.88
Death from all vascular causesSecondary60 (4.4%)44 (3.2%)0.75
Death from vascular causes + non-fatal strokeSecondary171 (12.4%)132 (9.7%)0.78
Major bleeding complication (any)Secondary53 (3.9%)35 (2.6%)0.67
All major ischaemic events (non-haemorrhagic vascular death, non-fatal ischaemic stroke, non-fatal MI)Secondary174 (12.6%)140 (10.3%)0.81
Vascular death + non-fatal stroke + non-fatal MISecondary192 (14.0%)149 (10.9%)0.78
First ischaemic strokeSecondary116 (8.4%)96 (7.0%)0.84
First cardiac event (fatal/non-fatal MI, sudden death, cardiac death)Secondary60 (4.4%)43 (3.2%)0.73
Primary composite (on-treatment analysis)Secondary--0.82
Updated meta-analysis with 5 prior trials (7795 patients, 1158 events) - vascular death/stroke/MISecondary--RR 0.82
Treatment discontinuationAdverse470/1363 (34%) aspirin+dipyridamole vs 184/1376 (13%) aspirin alone
Headache as reason for combination-arm discontinuationAdverse123/470 (26% of discontinuations); leading cause
Major bleeding (total)Adverse35 (2.6%) vs 53 (3.9%); HR 0.67 (95% CI 0.44-1.03)
Non-fatal extracranial major bleedAdverse21 vs 32
Fatal extracranial major bleedAdverse2 vs 0
Non-fatal intracranial major bleedAdverse9 vs 17
Fatal intracranial major bleedAdverse3 vs 4
Minor bleedingAdverse171 (12.5%) vs 168 (12.2%); RR 1.03 (95% CI 0.84-1.25)
Persistence at 1 yearAdverse77% combination vs 95% aspirin (of those still at risk)
Persistence at 3 yearsAdverse71% combination vs 89% aspirin
Persistence at 5 yearsAdverse66% combination vs 84% aspirin

Subgroup Analysis

No significant differences across prespecified subgroups: randomisation scheme (2-arm vs 3-arm), age (≤65 vs >65), sex, ischaemic heart disease history, cerebral ischaemia type (large vs small vessel), country (Asian vs non-Asian); smallest interaction p-value 0.18. Post-hoc subgroups (aspirin dose <40, 40-100, >100 mg; interval to randomisation <1 week, 1 week - 1 month, >1 month) also showed no material differences.


Criticisms

  • Open-label design (treatment not blinded) could bias notification and reporting of soft outcomes, although outcome adjudication was blinded and only clinically significant events were counted.
  • Wide latitude in aspirin dose (30-325 mg) with no fixed regimen; while intended to reflect real-world practice, it complicates direct comparison with fixed-dose trials such as ESPS-2 (aspirin 50 mg only).
  • 83% of combination-arm patients used extended-release dipyridamole; the other 17% used immediate-release (with different bioavailability), potentially diluting effect estimates.
  • Only ~30% of patients had large-vessel disease and only 66% had a completed minor stroke; two-thirds were randomised 1-6 months after the event, so ESPRIT does not address the acute high-risk window in which subsequent trials (CHANCE, POINT, THALES) focus.
  • Observed primary event rate on aspirin alone (4.8%/yr) was lower than the 6%/yr assumed for power, reflecting concomitant statin and antihypertensive use — leading to reduced absolute benefit and a wide NNT confidence interval (55-1006).
  • High discontinuation rate (34% vs 13%) driven by dipyridamole-induced headache limits real-world durability of benefit and biases the ITT estimate towards null; a titration strategy was not tested.
  • Twenty-four patients from one hospital were excluded before analysis for incomplete data, and follow-up was truncated at 11 patients from four other hospitals — small but non-random exclusions.
  • Classification of large vs small vessel disease relied on CT/clinical features (no routine diffusion-weighted MRI), so subgroup analyses by lesion type may have been misclassified in 10-20%.
  • The trial was not designed to detect a bleeding difference, so the unexpected numerical reduction in major bleeding with combination therapy is best interpreted as chance rather than a true safety signal.

Funding

Council of Singapore (NMRC/0702/2002, NMRC/0936/2005); European Commission (QLK6-CT-2002-02332); Janivo Foundation, Netherlands; French Ministry of Health (PHRC/1998); AEGON N V, Netherlands; Netherlands Heart Foundation (97.026); Thrombosis Foundation, Netherlands (2002.1); UK Stroke Association (24/96); University Medical Center Utrecht. No commercial/industry sponsor.

Based on: ESPRIT (Lancet, 2006)

Authors: Halkes PHA, van Gijn J, Kappelle LJ, ..., on behalf of the ESPRIT Study Group

Citation: Lancet 2006;367(9523):1665-1673. DOI: 10.1016/S0140-6736(06)68734-5

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