OBJECTIVES: Hemophilia is a bleeding disorder characterized by spontaneous bleedings due to deficiency of clotting factor. The most common site for bleeds is the joints which can be painful and may restrict daily activities. Current treatments are administered as subcutaneous (SC) injections or intravenous (IV) infusions with varying treatment frequencies. Treatment is lifelong, and it is therefore crucial to minimize treatment burden. The aim of this study was to investigate the treatment impact on HRQoL.

METHODS: Time trade-off methodology was used to estimate utilities through an online survey completed by the general population. Respondents evaluated health states as if they were living with hemophilia and treated themselves with prophylactic treatment. The following aspects were evaluated: frequency, device complexity (administration, preparation steps and time-use) and injection site reactions.

RESULTS: In total, 812, 739 and 703 respondents completed the survey in the UK, Canada and the US, respectively. The results suggest that device complexity is key. Using a pre-filled pen device for SC injections once every month (QM) was associated with a significant utility gain of 0.03 in all three countries when compared to a syringe for SC injections QM (p<0.001, CI 95% UK: 0.021-0.040, Canada: 0.020-0.040, US: 0.019-0.044). Furthermore, it was associated with a significant utility gain of 0.6 in the UK and of 0.4 in Canada and the US when compared to weekly IV treatment options (p<0.001, CI 95% UK: CI 95% 0.049-0.070, Canada: 0.026-0.056, US: 0.029-0.059). Additionally, the results indicate that less frequent treatments and treatments not associated with injection site reactions have the potential to increase HRQoL.

CONCLUSIONS: Different aspects of hemophilia treatment have an impact. Future treatments should focus on easy-to-use devices that require few preparation steps and a short time-use (including preparation and administration). This could lead to improvements in HRQoL among people with hemophilia.