title: Health-related quality-of-life treatments for severe haemophilia: utility measurements using the Standard Gamble technique.
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.
authors.affiliation: Faculty of Medicine, Hospital for Sick Children, Toronto, Ontario, Canada.
authors.forename: V S
authors.initials: VS
authors.lastname: Naraine
authors.forename: N A
authors.initials: NA
authors.lastname: Risebrough
authors.forename: P
authors.initials: P
authors.lastname: Oh
authors.forename: V S
authors.initials: VS
authors.lastname: Blanchette
authors.forename: S
authors.initials: S
authors.lastname: Lee
authors.forename: A-M
authors.initials: AM
authors.lastname: Stain
authors.forename: D
authors.initials: D
authors.lastname: Hedden
authors.forename: J M
authors.initials: JM
authors.lastname: Teitel
authors.forename: B M
authors.initials: BM
authors.lastname: Feldman
mesh_terms: D000328:Adult; D001294:Attitude to Health; D002408:Catheters, Indwelling; D003362:Cost-Benefit Analysis; D003661:Decision Support Techniques; D004334:Drug Administration Schedule; D005164:Factor IX / Q000627:therapeutic use; D005169:Factor VIII / Q000627:therapeutic use; D005260:Female; D006301:Health Services Needs and Demand; D006467:Hemophilia A / Q000188:drug therapy / Q000534:rehabilitation*; D002836:Hemophilia B / Q000188:drug therapy / Q000534:rehabilitation*; D006801:Humans; D008297:Male; D008875:Middle Aged; D010290:Parents / Q000523:psychology; D011788:Quality of Life; D018434:Sickness Impact Profile
chemical_list: D005169:Factor VIII; D005164:Factor IX