A cost evaluation of treatment alternatives for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil

Haemophilia. 2007 Sep;13(5):462-9. doi: 10.1111/j.1365-2516.2007.01522.x.

Abstract

The first-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil is currently activated prothrombin complex concentrate (aPCC), with recombinant activated factor VII (rFVIIa) used as second-line therapy or as a last resort. The aim of this study was to determine the cost and effectiveness of these treatments from the perspective of the Brazilian National Health Service. A decision analysis model was constructed to assess total direct medical costs (including drug costs, costs of outpatient or inpatient care, ambulance transportation and cost of concomitant medications) of first-line treatment with aPCC or rFVIIa. Clinical outcome and resource utilization data were obtained both retrospectively and prospectively and validated by the consensus of an expert panel of Brazilian haematologists. A total of 103 bleeds in 25 patients were included in the analysis. rFVIIa resolved bleeds more quickly (4.4 h) than aPCC (62.6 h) and was more effective (100% vs. 56.7% respectively). Mean total direct medical costs (from initiation to cessation of bleed) were estimated to be US$13 500 (aPCC) and US$7590 (rFVIIa). Extensive sensitivity analyses confirmed the cost-effectiveness of rFVIIa. Compared with aPCC, rFVIIa was more effective and less expensive when used as first-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil. rFVIIa should be considered a first-line treatment for the management of these patients.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Brazil
  • Child
  • Cohort Studies
  • Cost of Illness
  • Factor VII / economics
  • Factor VII / therapeutic use*
  • Factor VIIa
  • Female
  • Hemophilia A / drug therapy*
  • Hemophilia A / economics
  • Hemorrhage / economics
  • Hemorrhage / prevention & control*
  • Humans
  • Male
  • Prospective Studies
  • Recombinant Proteins / economics
  • Recombinant Proteins / therapeutic use
  • Retrospective Studies
  • Treatment Outcome

Substances

  • Recombinant Proteins
  • Factor VII
  • recombinant FVIIa
  • Factor VIIa