INTRODUCTION: An evolving hemophilia treatment landscape provides new possibilities for previously unattainable lifestyles.
AIM: We sought to understand how people with hemophilia (PwH) and their caregivers value potential benefits of novel prophylactic treatments. We conducted a discrete-choice experiment (DCE) to quantify preferences for features of hemophilia treatments among adults and caregivers of children with hemophilia. A best-worst scaling (BWS) exercise measured the perceived burden of treatment administration features.
METHODS: A cross-sectional, web-based survey was administered to male adults (≥18 years) and caregivers of male children (≤17 years) living with hemophilia in the United States. Respondents evaluated 8 pairs of hypothetical hemophilia treatment profiles defined by 6 attributes in the DCE and 15 features in the BWS.
RESULTS: In the DCE, both adults with hemophilia (n=151) and caregivers (n=151) prioritized avoiding the risk of developing inhibitor/ anti-drug antibodies and treatments that allowed for a more active life. They placed lower priority on reducing the number of spontaneous bleeding episodes, route and frequency of administration, and avoiding the risk of hospitalization due to adverse events. The BWS documented the burdensomeness of IV infusions and medications that require mixing and refrigeration.
CONCLUSION: PwH and caregivers prefer treatments that enable a more active lifestyle with a lower risk of inhibitor development. Both groups valued the ability to lead an active life over reducing spontaneous bleeding, with caregivers placing the most weight on this attribute. As new treatments expand possibilities, healthcare professionals and PwH should continue to share decision-making, incorporating clinical judgment and individual’s preferences.
