Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome

Haemophilia. 2002 Nov;8(6):745-52. doi: 10.1046/j.1365-2516.2002.00695.x.

Abstract

A multicentre study was performed to compare clotting factor use and outcome between on-demand and prophylactic treatment strategies for patients with severe haemophilia. Data on treatment and outcome of 49 Dutch patients with severe haemophilia, born 1970-80, primarily treated with prophylaxis, were compared with those of 106 French patients, who were primarily treated on demand. Dutch patients received intermediate dose prophylaxis, for a median duration of 12.7 years. Patients primarily treated with prophylaxis had fewer joint bleeds per year (median 2.8 vs. 11.5), a higher proportion of patients without joint bleeds (29% vs. 9%), lower clinical scores (median 2.0 vs. 8.0), and less arthropathy as measured by the Pettersson score (median 7 points vs. 16 points). Mean annual clotting factor use was equal at 1,488 +/- 783 IU kg-1 year-1 (mean +/- standard deviation) for patients primarily treated with prophylaxis and 1,612 +/- 1,442 IU kg-1 year-1 for patients primarily treated on demand. These findings suggest that, compared with a primarily on-demand treatment strategy, a primarily prophylactic treatment strategy leads to better outcome at equal treatment costs in young adults with severe haemophilia.

Publication types

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

MeSH terms

  • Adult
  • Drug Administration Schedule
  • Factor IX / administration & dosage*
  • Factor IX / therapeutic use
  • Factor VIII / administration & dosage*
  • Factor VIII / therapeutic use
  • Hemarthrosis / etiology
  • Hemarthrosis / prevention & control*
  • Hemophilia A / complications
  • Hemophilia A / drug therapy*
  • Hemophilia B / complications
  • Hemophilia B / drug therapy*
  • Humans
  • Male
  • Retrospective Studies
  • Treatment Outcome

Substances

  • Factor VIII
  • Factor IX