Abstract

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.