Patient and Caregiver Preferences for Hemophilia A Treatment in Taiwan: A Discrete Choice Experiment.

Objectives: This study evaluated the treatment preferences among patients and caregivers for moderate or severe hemophilia A in Taiwan.

Methods: This cross-sectional study used a discrete choice experiment approach to assess treatment preferences among patients with hemophilia A and their caregivers. Respondents chose between 2 hypothetical treatments defined by 7 attributes including no bleeding episode, treated events of joint bleeding, long-term safety, type of treatment and risk of thromboembolic events, administration frequency, consumption route, and monitoring dosing options. Preference weights and relative importance (RI) of attributes were estimated using a hierarchical Bayesian logistic regression model.

Results: A total of 51 eligible respondents completed the discrete choice experiment (patients, 76.5%; caregivers, 23.5%). Most patients (70.6%) had severe hemophilia and 43.1% received prophylactic treatment for >10 years. Respondents valued "type of treatment and risk of thromboembolic events" as the most important factor (RI 26.2%; 95% CI 20.9-31.5) followed by "consumption route" (RI 25.8%; 95% CI 20.7-30.9) and "administration frequency" (RI 15.2%; 95% CI 12.0-18.4). "Monitoring dosing options" was the least important attribute (RI 6.3%; 95% CI 5.2-7.4). Respondents had 63% possibility to choose a treatment profile that has factor VIII product compared with nonfactor product.

Conclusions: Patients and caregivers valued "type of treatment and risk of thromboembolic events" as the most important driver in choosing a treatment for moderate or severe hemophilia A. This study provides important insights into patients' preferences and informs clinicians to consider patients' choices when prescribing a treatment.