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Original Investigation
June 21, 2017

Trends and Outcomes of Off-Label Use of Transcatheter Aortic Valve ReplacementInsights From the NCDR STS/ACC TVT Registry

Author Affiliations
  • 1Section of Cardiology, University of Washington, Seattle
  • 2Duke Clinical Research Institute, Durham, North Carolina
  • 3University of Colorado School of Medicine, Aurora
  • 4Cleveland Clinic, Cleveland, Ohio
  • 5Michael E. DeBakey Veterans Affairs Medical Center, Houston, Texas
  • 6Baylor College of Medicine, Houston, Texas
  • 7Houston Methodist DeBakey Heart and Vascular Center, Houston, Texas
  • 8Weill Cornell Medical College, New York, New York
JAMA Cardiol. Published online June 21, 2017. doi:10.1001/jamacardio.2017.1685
Key Points

Question  What are the frequency and outcomes of the off-label use of transcatheter aortic valve replacement (TAVR) in the United States?

Findings  In this registry-based study, off-label TAVR was used in 9.5% of patients, with a cumulative survival rate of 74.4% at 1 year. In-hospital, 30-day, and 1-year mortality were higher among patients receiving off-label TAVR and was statistically significant; after adjustment, 1-year mortality was similar to that among patients receiving TAVR for on-label indications.

Meaning  Transcatheter aortic valve replacement may be a therapeutic option for patients with off-label indications whose conditions are inoperable or for high-risk operative candidates.

Abstract

Importance  Transcatheter aortic valve replacement (TAVR) was approved by the US Food and Drug Administration for severe aortic stenosis in patients who cannot undergo surgery and for patients at high operative risk. Use of TAVR for off-label indications has not been previously reported.

Objective  To evaluate patterns and adverse outcomes of off-label use of TAVR in US clinical practice.

Design, Setting and Participants  Patients receiving commercially funded TAVR in the United States are included in the Transcatheter Valve Therapy Registry. A total of 23 847 patients from 328 sites performing TAVR between November 9, 2011, and September 30, 2014, were assessed for this study. Off-label TAVR was defined as TAVR in patients with known bicuspid valve, moderate aortic stenosis, severe mitral regurgitation, severe aortic regurgitation, or subaortic stenosis. Data were linked with the Centers for Medicare & Medicaid Services for 15 397 patients to evaluate 30-day and 1-year outcomes.

Exposure  Off-label use of TAVR.

Main Outcomes and Measures  Frequency of off-label TAVR use and the association with in-hospital, 30-day, and 1-year adverse outcomes.

Results  Among the 23 847 patients in the study (11 876 women and 11 971 men; median age, 84 years [interquartile range, 78-88 years]), off-label TAVR was used in 2272 patients (9.5%). In-hospital mortality was higher among patients receiving off-label TAVR than those receiving on-label TAVR (6.3% vs 4.7%; P < .001), as was 30-day mortality (8.5% vs 6.1%; P < .001) and 1-year mortality (25.6% vs 22.1%; P = .001). Adjusted 30-day mortality was higher in the off-label group (hazard ratio, 1.27; 95% CI, 1.04-1.55; P = .02), while adjusted 1-year mortality was similar in the 2 groups (hazard ratio, 1.11; 95% CI, 0.98-1.25; P = .11). The median rate of off-label TAVR use per hospital was 6.8% (range, 0%-34.7%; interquartile range, 3.4%-12.1%), with hospitals in the highest tertile of off-label use associated with increased 30-day adverse cardiovascular events compared with the lowest tertile. However, this difference was not observed in adjusted 30-day or 1-year outcomes.

Conclusions and Relevance  Approximately 1 in 10 patients in the United States have received TAVR for an off-label indication. After adjustment, 1-year mortality was similar in these patients to that in patients who received TAVR for an on-label indication. These results reinforce the need for additional research on the efficacy of off-label TAVR use.

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