Cost-effectiveness of using recombinant activated factor VII as an off-label rescue treatment for critical bleeding requiring massive transfusion

摘要

Background: Recombinant activated factor VII (rFVIIa) is widely used as an off-label rescue treatment for patients with nonhemophilic critical bleeding. Study Design and Methods: Using data from the intensive care unit, transfusion service, and death registry, the long-term survival after using rFVIIa and the associated cost per life-year gained in a consecutive cohort of patients with critical bleeding requiring massive transfusion (10 red blood cell [RBC] units in 24 hr) were assessed. rFVIIa was only used as a lifesaving treatment when conventional measures had failed. Results: Of the 353 patients with critical bleeding requiring massive transfusion, 81 (23%) required rFVIIa as a lifesaving rescue treatment. The patients requiring rFVIIa received a greater number of transfusions (number of units: RBCs, 18 vs. 12; fresh-frozen plasma, 16 vs. 10; platelets, 4 vs. 2; p < 0.001) and had a shorter survival time (24 months vs. 33 months; p = 0.002) than those who did not require rFVIIa. The total cost per life-year gained of massive transfusion and incremental cost of rFVIIa as a lifesaving treatment were US$1,148,000 (￡711,760; 95% confidence interval [CI], US$825,000-US$1,471,000) and US$736,000 (￡456,320; 95% CI, US$527,000-US$945,000), respectively. The incremental costs of rFVIIa increased with severity of illness and transfusion requirement and were greater than the usual acceptable cost-effective limit (