Comparative survival and cost-effectiveness of advanced therapies for end-stage heart failure

Background: Treatment options for end-stage heart failure include inotrope-dependent medical therapy, orthotopic heart transplantation (OHT), left ventricular assist device (LVAD) as destination therapy or bridge to transplant.

Methods and results: We developed a state-transition model to simulate 4 treatment options and associated morbidity and mortality. Transition probabilities, costs, and utilities were estimated from published sources. Calculated outcomes included survival, quality-adjusted life-years, and incremental cost-effectiveness. Sensitivity analyses were performed on model parameters to test robustness. Average life expectancy for OHT-eligible patients is estimated at 1.1 years, with 39% surviving to 1 year. OHT with a median wait time of 5.6 months is estimated to increase life expectancy to 8.5 years, and costs <$100,000/quality-adjusted life-year gained, relative to inotrope-dependent medical therapy. Bridge to transplant-LVAD followed by OHT further is estimated to increase life expectancy to 12.3 years, for $226,000/quality-adjusted life-year gained versus OHT. Among OHT-ineligible patients, mean life expectancy with inotrope-dependent medical therapy is estimated at 9.4 months, with 26% surviving to 1 year. Patients who instead received destination therapy-LVAD are estimated to live 4.4 years on average from extrapolation of recent constant hazard rates beyond the first year. This strategy costs $202,000/quality-adjusted life-year gained, relative to inotrope-dependent medical therapy. Patient's age, time on wait list, and costs associated with care influence outcomes.

Conclusions: Under most scenarios, OHT prolongs life and is cost effective in eligible patients. Bridge to transplant-LVAD is estimated to offer >3.8 additional life-years for patients waiting ≥6 months, but does not meet conventional cost-effectiveness thresholds. Destination therapy-LVAD significantly improves life expectancy in OHT-ineligible patients. However, further reductions in adverse events or improved quality of life are needed for destination therapy-LVAD to be cost effective.