title: Cost-effectiveness of recombinant activated factor VII vs. plasma-derived activated prothrombin complex concentrate in the treatment of mild-to-moderate bleeding episodes in patients with severe haemophilia A and inhibitors in Spain.
abstract: Several analyses have shown that recombinant activated factor VII (rFVIIa) is a cost-effective intervention compared with plasma-derived activated prothrombin complex concentrate (pd-aPCC) for the on-demand treatment of mild-to-moderate bleeds in haemophilia patients with inhibitors. The aim of the study was to assess the cost-effectiveness of rFVIIa vs. pd-aPCC in the treatment of bleeding episodes in severe haemophilia A patients with inhibitors in Spain. A decision analytic model was designed to evaluate the costs and clinical outcomes of using rFVIIa or pd-aPCC to treat mild-to-moderate joint bleeds in children (≤14 years old) and adults with inhibitors. Data were obtained from a published meta-analysis and a panel of haemophilia experts. The analysis was conducted from the perspective of the Spanish National Healthcare System. One-way sensitivity analyses were performed to assess the impact of model assumptions on study results. In the Treur meta-analysis, rFVIIa resulted in cumulative joint bleed resolution of 88% and 95% after 24 and 36 h, respectively, compared with 62% and 76%, respectively, with pd-aPCC (Treur et al. Haemophilia 2009; 15: 420-36). Here, the mean cost per bleed was estimated at €8473 and €15 579 in children and adults treated with rFVIIa, vs. €8627 and €15 677 in children and adults treated with pd-aPCC. rFVIIa treatment was found to be the dominating option (cheaper and more effective). The one-way sensitivity analysis also confirmed that rFVIIa was less costly than pd-aPCC. The model suggests that rFVIIa is a cost-effective option compared with pd-aPCC for the treatment of mild-to-moderate bleeding episodes in a Spanish setting.
authors.affiliation: Hospital Universitario La Paz, Autonoma University, Madrid, Spain.
authors.forename: V
authors.initials: V
authors.lastname: Jimenez-Yuste
authors.forename: R
authors.initials: R
authors.lastname: Núñez
authors.forename: J A
authors.initials: JA
authors.lastname: Romero
authors.forename: B
authors.initials: B
authors.lastname: Montoro
authors.forename: B
authors.initials: B
authors.lastname: Espinós
mesh_terms: D000328:Adult; D019774:Blood Coagulation Factor Inhibitors / Q000097:blood*; D001779:Blood Coagulation Factors / Q000627:therapeutic use*; D002648:Child; D003029:Coagulants / Q000627:therapeutic use*; D003362:Cost-Benefit Analysis; D015942:Factor VIIa / Q000627:therapeutic use*; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D006470:Hemorrhage / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D011994:Recombinant Proteins / Q000627:therapeutic use; D012720:Severity of Illness Index; D013030:Spain
chemical_list: D019774:Blood Coagulation Factor Inhibitors; D001779:Blood Coagulation Factors; D003029:Coagulants; D011994:Recombinant Proteins; C103587:recombinant FVIIa; C065655:anti-inhibitor coagulant complex; D015942:Factor VIIa
keywords: Spain; congenital haemophilia with inhibitors; cost-effectiveness; plasma-derived activated prothrombin complex concentrate; recombinant activated factor VII