Health-related quality of life as outcome parameter in haemophilia treatment

Health-related quality of life (HRQoL) is increasingly used as an outcome parameter in haemophilia treatment. Currently, only generic HRQoL instruments are used, as disease-specific instruments for haemophilia are not available. The most widely used generic instruments are the descriptive SF-36 questionnaire and the EQ-5D questionnaire for assessment of utilities, ie preference-based HRQoL. Results of both instruments show significant correlation. All haemophilia prophylaxis studies using HRQoL as an outcome report a decreased HRQoL compared with the general population, a positive effect of prophylactic treatment and a negative effect of age. Generic instruments for measuring HRQoL are able to pick up differences between groups of patients with haemophilia and enable us to make comparisons across diseases. However, in order to establish the optimal treatment strategy for severe haemophilia, further information is needed on the long-term effects of different strategies on HRQoL. In addition, haemophilia-specific HRQoL instruments should be developed in order to provide detailed information for adjustment and evaluation of treatment in individual patients.