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RESPECT (Original 2013): Original Primary Publication: PFO Closure with Amplatzer Occluder vs Medical Therapy after Cryptogenic Stroke (Carroll et al., 2013)

This is the original 2013 RESPECT primary publication, DISTINCT from the 2017 long-term extension. In patients 18–60 with cryptogenic ischemic stroke and a PFO, did transcatheter closure with the Amplatzer PFO Occluder reduce recurrent ischemic stroke or early death vs medical therapy at median 2.1-year follow-up? Note: ITT primary NOT MET (HR 0.49, P=0.08 borderline); per-protocol HR 0.37 (P=0.03); as-treated HR 0.27 (P=0.007). The 2017 extended follow-up (see RESPECT long-term) ultimately converted the result.

Carroll JD et al. (NEJM 2013;368:1092-1100) · doi:10.1056/NEJMoa1301440 · 980 patients

Population

Included

  • Age 18–60 years
  • Cryptogenic ischemic stroke within 270 days before randomization
  • TEE-confirmed PFO
  • Mechanism workup excluded large-vessel arteriopathy, established cardioembolic source, intrinsic small-vessel disease, and arterial hypercoagulable state

Excluded

  • Identifiable competing stroke mechanism
  • Age outside 18–60
  • Inability to undergo randomization within 270 days of index stroke

Primary Outcome

Recurrent ischemic stroke or early death at median 2.1-year follow-up (intention-to-treat)

PFO Closure (Amplatzer)
1 / 100
Medical Therapy
3 / 100

Negligible absolute difference

Recurrent ischemic stroke at 2.1y (ITT)

Risk ratio HR 0.4995% CI 0.22–1.11p = 0.08

Safety

Serious adverse events at median 2.1y

PFO Closure (Amplatzer)

23%

Medical Therapy

21.6%

Serious adverse events occurred in 23.0% (closure) vs 21.6% (medical therapy) over the original median 2.1-year follow-up, no significant difference. Procedure-related serious events occurred in 4.2% of closure patients. Per the original publication, atrial fibrillation rate was NOT increased, a notable difference from CLOSURE-I, REDUCE, and CLOSE.

Trial Design

Type

  • Multicenter, randomized, open-label, controlled superiority trial with blinded endpoint adjudication
  • 1:1 randomization (PFO closure with Amplatzer : medical therapy)
  • Stratified randomization by site, planned antithrombotic regimen, and presence/absence of atrial septal aneurysm
  • 69 sites in US and Canada
  • Event-driven design with three pre-specified analyses: intention-to-treat, per-protocol, and as-treated
  • Original 2013 publication: primary analysis at median 2.1-year follow-up; ITT NOT MET
  • Extended follow-up at median 5.9y (Saver JL et al., NEJM 2017;377:1022; trial ID respect-trial) ultimately converted the result

Timeline

Enrolled August 2003 to December 2011; original primary analysis published NEJM 2013;368:1092 at median 2.1y

N

980

Enrollment

980 patients (499 closure / 481 medical therapy) at 69 US + Canadian sites. Enrolled August 2003 to December 2011. Median 2.1-year follow-up. Amplatzer PFO Occluder (St. Jude Medical). Original primary ITT analysis published NEJM 2013;368:1092 — borderline (P=0.08), NOT MET. The 2017 extended follow-up at median 5.9y (catalog entry respect-trial) converted the result (HR 0.55, P=0.046).

ClinicalTrials.gov

NCT00465270

Bedside Pearl

Original RESPECT (NEJM 2013;368:1092; DISTINCT from the 2017 long-term extension): ITT primary NOT MET at median 2.1y (HR 0.49, 95% CI 0.22–1.11, P=0.08, BORDERLINE). Per-protocol HR 0.37 (P=0.03) and as-treated HR 0.27 (P=0.007) favored closure but cannot substitute for ITT. The 2017 extended follow-up at median 5.9y (HR 0.55, P=0.046; catalog entry respect-trial) ultimately converted the result and underwrote FDA approval of the Amplatzer device.

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