On-demand vs. prophylactic treatment for severe haemophilia in Norway and Sweden: differences in treatment characteristics and outcome

Haemophilia. 2003 Sep;9(5):555-66. doi: 10.1046/j.1365-2516.2003.00817.x.

Abstract

Using an 11-year panel of 156 Norwegian and Swedish patients with severe haemophilia, and including retrospective case-book data from birth, we compared the differences in the haemophilia-related resource use between on-demand and prophylactic treatment. Patients treated on-demand had more surgery (arthrodeses, prostheses implantations and synovectomies) and more days lost from work. Median annual factor-concentrate consumption among adults (18+) was 211,000 IU [interquartile range (IQR) 154,000-268,000] or 3,024 IU kg-1 year(-1) for patients on prophylactic treatment and 55,000 IU (IQR 28,000-91,000) for on-demand patients (780 IU kg(-1) year(-1)). This was partly explained by the fact that the median dose per kg body weight was twice as great 28, (IQR 24-32) for prophylaxis compared with 14 (IQR 12-16) for on-demand. Prescribed dose per kg body weight was found to be an important factor explaining the variation in total annual factor-concentrate consumption per patient for both types of treatment. Other variables included in the panel-data regression analysis were the number of weeks on secondary prophylaxis for on-demand patients and age, body weight and type of haemophilia for children (0-17 years) on prophylaxis. Differences were consistently substantial and will affect both costs and benefits of the two treatment strategies.

Publication types

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

MeSH terms

  • Absenteeism
  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Drug Administration Schedule
  • Employment / statistics & numerical data
  • Factor IX / administration & dosage*
  • Factor IX / therapeutic use
  • Factor VIII / administration & dosage*
  • Factor VIII / therapeutic use
  • Hemarthrosis / prevention & control
  • Hemarthrosis / surgery
  • Hemophilia A / complications
  • Hemophilia A / drug therapy*
  • Hemophilia B / complications
  • Hemophilia B / drug therapy*
  • Hemorrhage / prevention & control
  • Hospitalization / statistics & numerical data
  • Humans
  • Infant
  • Male
  • Middle Aged
  • Norway
  • Retrospective Studies
  • Sweden
  • Treatment Outcome

Substances

  • Factor VIII
  • Factor IX