Cost-effectiveness of using recombinant activated factor VII as an off-label rescue treatment for critical bleeding requiring massive transfusion.
BACKGROUND
Recombinant activated factor VII (rFVIIa) is widely used as an off-label rescue treatment for patients with nonhemophilic critical bleeding.


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 (<US$100,000 per life-year) for most patients with critical bleeding.


CONCLUSIONS
As a lifesaving treatment for critical bleeding, the incremental cost of rFVIIa was high. Careful patient selection is critical to balance the potential benefits of rFVIIa in an individual patient against the cost to the community.
Department of Intensive Care Medicine, Royal Perth Hospital, Perth, Western Australia, Australia. kwok.ho@health.wa.gov.au
Kwok M
KM
Ho
Edward
E
Litton
D000328:Adult; D000368:Aged; D001771:Blood Banks / Q000191:economics / Q000706:statistics & numerical data; D015331:Cohort Studies; D003131:Combined Modality Therapy / Q000191:economics / Q000401:mortality; D003362:Cost-Benefit Analysis; D003422:Critical Care / Q000191:economics / Q000379:methods / Q000706:statistics & numerical data; D017707:Erythrocyte Transfusion / Q000191:economics / Q000401:mortality; D015942:Factor VIIa / Q000191:economics* / Q000627:therapeutic use*; D005260:Female; D006470:Hemorrhage / Q000188:drug therapy* / Q000191:economics* / Q000401:mortality; D006801:Humans; D008297:Male; D008875:Middle Aged; D056687:Off-Label Use / Q000191:economics* / Q000706:statistics & numerical data; D011994:Recombinant Proteins / Q000191:economics / Q000627:therapeutic use; D012042:Registries / Q000706:statistics & numerical data; D016879:Salvage Therapy / Q000191:economics / Q000379:methods / Q000401:mortality; D012720:Severity of Illness Index; D016019:Survival Analysis
D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa