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Outcomes of Off-Label Use of TAVR
Study Questions:
What are the frequency and outcomes of the off-label use of transcatheter aortic valve replacement (TAVR) in the United States?
Methods:
The investigators used data from the Society of Thoracic Surgeons (STS) and the American College of Cardiology (ACC) Transcatheter Valve Therapy (TVT) Registry. Patients receiving commercially funded TAVR in the United States are included in this registry. A total of 23,847 patients from 328 sites performing TAVR between November 9, 2011, and September 30, 2014, were assessed. 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 and Medicaid Services for 15,397 patients to evaluate 30-day and 1-year outcomes. The main outcomes and measures were 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 2,272 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 < 0.001), as was 30-day mortality (8.5% vs. 6.1%; p < 0.001) and 1-year mortality (25.6% vs. 22.1%; p = 0.001). Adjusted 30-day mortality was higher in the off-label group (hazard ratio [HR], 1.27; 95% confidence interval [CI], 1.04-1.55; p = 0.02), while adjusted 1-year mortality was similar in the two groups (HR, 1.11; 95% CI, 0.98-1.25; p = 0.11). 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:
The authors reported that after adjustment, 1-year mortality was similar in off-label patients to that in patients who received TAVR for an on-label indication.
Perspective:
This national registry analysis suggests that approximately 1 in 10 patients received TAVR for an off-label indication. Although, off-label TAVR use was associated with higher in-hospital, 30-day, and 1-year mortality rates compared with on-label TAVR use, after adjustment, 1-year mortality was similar in the two groups. It should be noted that lack of differences in statistically adjusted outcomes between ‘on-label’ and ‘off-label’ use should not be a basis for labeling or new clinical recommendation. A randomized controlled trial directly comparing TAVR with surgery among specific subsets of off-label populations is needed to provide convincing evidence, and significant ‘off-label’ use captured in registries such as this should motivate such prospective randomized studies. Finally, understanding whether off-label TAVR use carries acceptable risks and has meaningful benefits requires data on patients with similar comorbidities who did not undergo TAVR, including those who underwent valve surgery or received no intervention.
Keywords: Aortic Valve Insufficiency, Aortic Valve Stenosis, Cardiac Surgical Procedures, Constriction, Pathologic, Geriatrics, Heart Valve Diseases, Hospital Mortality, Mitral Valve Insufficiency, Off-Label Use, STS/ACC TVT Registry, Transcatheter Aortic Valve Replacement, Treatment Outcome
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