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DEFENSE-PFO Trial: Device Closure vs Medication for Cryptogenic Stroke Patients With High-Risk PFO Anatomy (Lee et al., 2018)

In patients with cryptogenic ischemic stroke and HIGH-RISK PFO anatomy (atrial septal aneurysm, septal hypermobility with phasic excursion ≥10 mm, or PFO size ≥2 mm), does transcatheter PFO closure reduce 2-year stroke / vascular death / TIMI major bleeding compared with medication-only therapy? Confirmed the morphology-enrichment hypothesis operationalized by CLOSE (2017).

Lee PH et al. (JACC 2018;71:2335-2342) · doi:10.1016/j.jacc.2018.02.046 · 120 patients

Population

Included

  • Cryptogenic ischemic stroke
  • PFO with HIGH-RISK anatomy by TEE: atrial septal aneurysm, septal hypermobility (phasic septal excursion into either atrium ≥10 mm), or PFO size (maximum separation of septum primum from secundum) ≥2 mm
  • Adequate workup excluding alternative stroke etiologies

Excluded

  • Identifiable competing stroke mechanism
  • PFO without any of the three high-risk anatomic features
  • Contraindication to PFO closure or to antiplatelet/anticoagulation therapy

Primary Outcome

2-year composite of stroke, vascular death, or TIMI-defined major bleeding

PFO ClosureBetter outcome
0 / 100
Medication-only
12 / 100

Negligible absolute difference

Primary composite at 2 years (KM)

Risk ratio 0/60 vs 6/6095% CI —–—p = 0.013
NNT8to prevent one primary endpoint event over 2 years

Safety

Atrial fibrillation (procedure-related)

PFO Closure

3.3%

Medication-only

0%

Procedure-related nonfatal atrial fibrillation occurred in 2 of 60 closure patients (3.3%); pericardial effusion in 1 (1.7%); pseudoaneurysm in 1 (1.7%). No comparable procedural complications in the medication-only arm. Source: Lee PH et al., JACC 2018;71:2335-2342.

Trial Design

Type

  • Investigator-initiated, multicenter, randomized, open-label superiority trial with blinded endpoint adjudication
  • 1:1 randomization (PFO closure : medication-only)
  • 2 sites in Korea (Asan Medical Center, Seoul St Mary's Hospital)
  • HIGH-RISK PFO anatomy required (atrial septal aneurysm, hypermobility, or size ≥2 mm)
  • Small sample size (N=120); confirmed morphology-enrichment hypothesis from the 2017 NEJM cluster

Timeline

Enrolled September 2011 to October 2017; 2-year follow-up; published JACC 2018

N

120

Enrollment

120 Korean patients (60 closure / 60 medication-only) at 2 sites (Asan Medical Center, Seoul St Mary's Hospital). Enrolled September 2011 to October 2017. 2-year follow-up. High-risk PFO anatomy required (atrial septal aneurysm, septal hypermobility ≥10 mm, or PFO size ≥2 mm). Published JACC 2018.

ClinicalTrials.gov

NCT01550588

Bedside Pearl

DEFENSE-PFO (JACC 2018): in 120 Korean patients with cryptogenic stroke and HIGH-RISK PFO anatomy (atrial septal aneurysm, hypermobility ≥10 mm, or PFO size ≥2 mm), closure beat medical therapy on the 2-year composite of stroke / vascular death / TIMI major bleeding (0/60 vs 6/60, KM 2-year rate 12.9%, log-rank P=0.013). 2-year ischemic stroke 0% vs 10.5% (P=0.023). Confirms the morphology-enrichment hypothesis from CLOSE and RESPECT long-term subgroup.

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