Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen

Haemophilia. 2002 Nov;8(6):753-60. doi: 10.1046/j.1365-2516.2002.00694.x.

Abstract

A multicentre study was performed in Sweden and the Netherlands, comparing effects of two prophylactic regimens in 128 patients with severe haemophilia, born 1970-90. 42 Swedish patients (high-dose prophylaxis), were compared with 86 Dutch patients (intermediate-dose prophylaxis). Patients were evaluated at the date of their last radiological score according to Pettersson. Annual clotting factor consumption and bleeding frequency were registered for a period of three years before evaluation. Patients in the high-dose group were younger at evaluation (median 15.2 vs. 17.9 years), started prophylaxis earlier (median 2 vs. 5 years), and used 2.19 times more clotting factor kg-1 year-1. Patients treated with high-dose prophylaxis had fewer joint bleeds (median 0.3 year-1 vs. 3.3 year-1) and the proportion of patients without arthropathy as measured by the Pettersson score was higher (69% vs. 32%), however, the age-adjusted difference in scores (median 0 points vs. 4 points) was small and at present not statistically significant. Clinical scores and quality of life were similar. These findings suggest that, compared with intermediate-dose prophylaxis, high-dose prophylaxis significantly increases treatment costs and reduces joint bleeds over a period of 3 years, but only slightly reduces arthropathy after 17 years of follow-up.

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Dose-Response Relationship, Drug
  • Factor IX / administration & dosage*
  • Factor IX / therapeutic use
  • Factor VIII / administration & dosage*
  • Factor VIII / therapeutic use
  • Follow-Up Studies
  • Hemarthrosis / etiology
  • Hemarthrosis / prevention & control
  • Hemophilia A / complications
  • Hemophilia A / drug therapy*
  • Hemophilia B / complications
  • Hemophilia B / drug therapy*
  • Humans
  • Male
  • Quality of Life
  • Treatment Outcome

Substances

  • Factor VIII
  • Factor IX