Health-related quality-of-life treatments for severe haemophilia: utility measurements using the Standard Gamble technique

Haemophilia. 2002 Mar;8(2):112-20. doi: 10.1046/j.1365-2516.2002.00591.x.

Abstract

Prophylaxis for haemophilia improves outcomes, but at a substantial cost. Cost-utility analysis balances improvements seen in health-related quality of life (HRQoL) against costs, with the purpose of aiding healthcare decision-making. This analysis uses a measure of HRQoL known as utility. The objective of this study was to measure HRQoL (utility) values for states of health that result from on-demand therapy or prophylaxis. The HRQoL for different health states (including target joint bleeding, different intensities of prophylaxis, and indwelling intravenous catheters [ports]) was measured for healthy adults (n=30), parents of haemophilic children (n=30), and adults with haemophilia (n=28). Parents and patients rated health states similarly. Healthy adults gave the lowest ratings. The following rank, in order of HRQoL, was obtained: prophylaxis (low > medium > high) > on-demand therapy > prophylaxis with port> prophylaxis with infected port > on-demand therapy with development of a target joint. We conclude that: (1) haemophilia and its treatment reduce HRQoL; (2) prophylaxis is preferred to on-demand therapy; (3) intravenous ports substantially reduce HRQoL; (4) and an intravenous port to provide prophylaxis is preferable to on-demand therapy if a target joint develops.

MeSH terms

  • Adult
  • Attitude to Health
  • Catheters, Indwelling
  • Cost-Benefit Analysis
  • Decision Support Techniques
  • Drug Administration Schedule
  • Factor IX / therapeutic use
  • Factor VIII / therapeutic use
  • Female
  • Health Services Needs and Demand
  • Hemophilia A / drug therapy
  • Hemophilia A / rehabilitation*
  • Hemophilia B / drug therapy
  • Hemophilia B / rehabilitation*
  • Humans
  • Male
  • Middle Aged
  • Parents / psychology
  • Quality of Life*
  • Sickness Impact Profile*

Substances

  • Factor VIII
  • Factor IX