Health-related quality-of-life treatments for severe haemophilia: utility measurements using the Standard Gamble technique.
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.
Naraine
V S
VS
Faculty of Medicine, Hospital for Sick Children, Toronto, Ontario, Canada.
Risebrough
N A
NA
Oh
P
P
Blanchette
V S
VS
Lee
S
S
Stain
A-M
AM
Hedden
D
D
Teitel
J M
JM
Feldman
B M
BM
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
D005169:Factor VIII; D005164:Factor IX