OBJECTIVES: This study aims to assess the treatment preferences among moderate or severe hemophilia A (HA) patients based on different features of current hemophilia therapeutics.

METHODS: This multicenter, cross-sectional study was conducted between April 2021-April 2022. A discrete-choice experiment (DCE) method was used to examine the treatment preferences of patients (adults aged ≥20 years) or caregivers (patients aged <20 years). Respondents’ willingness was assessed in terms of accepting trade-offs among hypothetical treatment profiles described by seven attributes (Bleeding episode, Treated events of joint bleeding, Long-term safety, Treatment type and risk of thromboembolic events, Administration frequency, Consumption route, and Dosing monitoring options) of different levels. Choice data were analyzed using Hierarchical Bayesian logistic regression model and relative importance (RI) was calculated based on the differences of utility scores to determine the treatment preferences.

RESULTS: Among 51 study respondents recruited, 76% (n=39) were patients themselves. Hemophilia patients, as reported by the respondents, were mostly males (98%, n=50), 71% (n=36) had severe HA and 29% (n=15) had moderate HA. Among HA patients, 43% (n=22) had college degree and 29% (n=15) were aged 20-29 years. Twenty-two (43%) patients received prophylactic treatment for over 10 years, while 7 (14%) reported not having received prophylactic treatment. For caregivers, half of them were males (n=6) and 42% (n=5) aged 40-49 years. “Treatment type and risk of thromboembolic events” (RI=26.2%) was regarded by patients and caregivers as the most important attribute, followed by “Consumption route” (RI = 25.8%). Within “treatment type and risk of thromboembolic events”, respondents preferred “Factor VIII product with no risk of thromboembolic events” (utility=1.18)” over “non-Factor VIII product with risk of thromboembolic events” (utility=-1.18). “Monitoring dosing options” was the least important consideration when switching treatment (RI=6.3%).

CONCLUSIONS: This study provides an important insight towards understanding patient preferences when considering therapeutic options for HA.