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.