title: Clinical outcomes with on-label and off-label use of the transcatheter heart valve in the United States.
abstract: OBJECTIVE
  We explored the efficacy, safety, and clinical consequences of on-label and off-label transcatheter aortic valve replacement (TAVR) in the real-world setting.
  
  
  BACKGROUND
  The transcatheter heart valve (THV) was initially approved only for transfemoral (TF) delivery (on-label use) during TAVR in inoperable patients with severe aortic stenosis (AS). Because of lack of alternative options in TAVR-eligible patients with inadequate TF access, other routes have been utilized for THV implantation (off-label use), outcomes of which were previously unknown.
  
  
  METHODS
  Consecutive patients with severe inoperable AS who underwent clinical TAVR at our site were enrolled in a prospective database. Fifty subjects underwent TF-TAVR (on-label group), while non-TF routes were utilized in 60 subjects (off-label group). Procedural events, 30-day clinical outcomes, and 1-year all-cause mortality data were analyzed.
  
  
  RESULTS
  Technical device success was similar between on-label and off-label groups (88% vs. 87%, respectively; P = 0.92), as was the incidence of procedural complications and 30-day clinical events. The on-label group had lower 1-year all-cause death rate (12%) compared to the off-label group (32%; P = 0.02). The 1-year all-cause mortality in the off-label group was comparable to published clinical trial and registry data on TAVR, and appeared lower than historical outcomes with conservative medical therapy.
  
  
  CONCLUSIONS
  On-label use of the THV in the real-world setting was associated with favorable survival outcomes compared to off-label TAVR and historical data. Off-label use of the THV appeared to be safe and effective when used in select patients with inoperable AS who are not eligible for TAVR via TF approach.
authors.affiliation: The Multi-Disciplinary Structural Heart Disease Program of the University of Miami, Miller School of Medicine, Miami, Florida.
authors.forename: Joel A
authors.initials: JA
authors.lastname: Lardizabal
authors.forename: Conrad J
authors.initials: CJ
authors.lastname: Macon
authors.forename: Brian P
authors.initials: BP
authors.lastname: O'Neill
authors.forename: Vikas
authors.initials: V
authors.lastname: Singh
authors.forename: Claudia A
authors.initials: CA
authors.lastname: Martinez
authors.forename: Carlos
authors.initials: C
authors.lastname: Alfonso
authors.forename: Mauricio G
authors.initials: MG
authors.lastname: Cohen
authors.forename: Donald B
authors.initials: DB
authors.lastname: Williams
authors.forename: William W
authors.initials: WW
authors.lastname: O'Neill
authors.forename: Alan W
authors.initials: AW
authors.lastname: Heldman
mesh_terms: D000369:Aged, 80 and over; D001024:Aortic Valve Stenosis / Q000401:mortality / Q000601:surgery*; D002423:Cause of Death / Q000639:trends; D005260:Female; D005500:Follow-Up Studies; D006350:Heart Valve Prosthesis; D006801:Humans; D008297:Male; D011446:Prospective Studies; D018570:Risk Assessment; D012307:Risk Factors; D015996:Survival Rate / Q000639:trends; D065467:Transcatheter Aortic Valve Replacement / Q000379:methods*; D016896:Treatment Outcome; D014481:United States / Q000453:epidemiology
keywords: aortic stenosis; structural heart intervention; valvular heart disease