A cost minimization model for the treatment of minor bleeding episodes in patients with haemophilia A and high-titre inhibitors

Haemophilia. 2005 May;11(3):261-9. doi: 10.1111/j.1365-2516.2005.01098.x.

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.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Autoantibodies / immunology
  • Blood Coagulation Factors / administration & dosage
  • Blood Coagulation Factors / economics
  • Blood Coagulation Factors / therapeutic use*
  • Child
  • Drug Administration Schedule
  • Factor VII / administration & dosage
  • Factor VII / economics
  • Factor VII / therapeutic use
  • Factor VIII / immunology
  • Factor VIIa
  • Health Care Costs
  • Hemarthrosis / drug therapy*
  • Hemarthrosis / economics
  • Hemarthrosis / etiology
  • Hemophilia A / complications
  • Hemophilia A / drug therapy*
  • Hemophilia A / economics
  • Home Care Services / economics
  • Humans
  • Models, Economic
  • Recombinant Proteins / administration & dosage
  • Recombinant Proteins / economics
  • Recombinant Proteins / therapeutic use

Substances

  • Autoantibodies
  • Blood Coagulation Factors
  • Recombinant Proteins
  • prothrombin complex concentrates
  • Factor VII
  • Factor VIII
  • recombinant FVIIa
  • Factor VIIa