title: A cost minimization model for the treatment of minor bleeding episodes in patients with haemophilia A and high-titre inhibitors.
abstract: Treatment of acute bleeding episodes in patients with haemophilia A and inhibitory antibodies to factor VIII (FVIII) most often involves the use of bypassing haemostatic agents, such as activated prothrombin complex concentrates (aPCC) or recombinant factor VIIa (rFVIIa). We constructed a cost minimization model to compare the costs of initial treatment with aPCC vs. rFVIIa in the home treatment of minor bleeding episodes. We developed a clinical scenario describing such a case and presented it to a panel of US haemophilia specialists. For each product class, we asked panellists to provide dosing regimens required to achieve complete resolution of a minor haemarthrosis in a child with high-titre inhibitors, and for the probabilities of success at two time points (8-12 and 24 h). Consensus among the panellists was refined by a second round of the process, and the median values resulting were used as inputs to a decision analysis model. Sensitivity analyses were conducted to determine threshold values for key variables. The base case model found that initial treatment with aPCC would result in a mean cost per episode of 21 000 dollars, compared with 33 400 dollars for initial treatment with rFVIIa. Sensitivity analyses over a range of clinically plausible values for cost, dosing, and efficacy did not change the selection of aPCC as the dominant strategy.
authors.affiliation: Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital and Harvard Medical School, Boston, MA 02120, USA.
authors.forename: K G
authors.initials: KG
authors.lastname: Putnam
authors.forename: R L
authors.initials: RL
authors.lastname: Bohn
authors.forename: B M
authors.initials: BM
authors.lastname: Ewenstein
authors.forename: W C
authors.initials: WC
authors.lastname: Winkelmayer
authors.forename: J
authors.initials: J
authors.lastname: Avorn
mesh_terms: D001323:Autoantibodies / Q000276:immunology; D001779:Blood Coagulation Factors / Q000008:administration & dosage / Q000191:economics / Q000627:therapeutic use*; D002648:Child; D004334:Drug Administration Schedule; D005167:Factor VII / Q000008:administration & dosage / Q000191:economics / Q000627:therapeutic use; D005169:Factor VIII / Q000276:immunology; D015942:Factor VIIa; D017048:Health Care Costs; D006395:Hemarthrosis / Q000188:drug therapy* / Q000191:economics / Q000209:etiology; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy* / Q000191:economics; D006699:Home Care Services / Q000191:economics; D006801:Humans; D018803:Models, Economic; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000191:economics / Q000627:therapeutic use
chemical_list: D001323:Autoantibodies; D001779:Blood Coagulation Factors; D011994:Recombinant Proteins; C025667:prothrombin complex concentrates; D005167:Factor VII; D005169:Factor VIII; C103587:recombinant FVIIa; D015942:Factor VIIa