Accepted for/Published in: JMIR Public Health and Surveillance
Date Submitted: Jan 17, 2023
Date Accepted: Jun 17, 2023
Warning: This is an author submission that is not peer-reviewed or edited. Preprints - unless they show as "accepted" - should not be relied on to guide clinical practice or health-related behavior and should not be reported in news media as established information.
Quantifying Benefit-Risk Preferences of Patient with Hemophilia A for Prophylactic Treatment: A Stated-Preference Study
ABSTRACT
Background:
Clinical trials have demonstrated that prophylactic treatment is beneficial to patients with hemophilia A (PwHA) without inhibitors. However, patients have poor adherence to prophylaxis in mainland China. Eliciting the patients' benefit-risk preferences is beneficial to promote adherence.
Objective:
To evaluate which treatment attributes PwHA prioritized and maximum acceptable risk (MAR) in prophylaxis.
Methods:
: Patients were recruited from seven regions in China to complete the online survey. Preference was assessed using discrete choice experiment (DCE) comprised of four attributes each with three levels, including annual bleeding rate, development of inhibitors, dosing frequency and dosing mode. Data were analysed using mixed logit model (MXL) with relative importance (RI) and MAR calculated. The sensitivity analysis was explored by conditional logit model (CLM).
Results:
A total of 113 patients were included in the analysis. More than half of them (58.41%) were severe hemophilia A (HA). All attributes had statistical significance (P < .05). Patients most valued the annual bleeding rate (RI:37.21%, 95%CI:[0.33,0.41]), followed by the development of inhibitors (RI:30.65%, 95%CI:[0.26,0.35]), and the dosing frequency (RI: 17.07%, 95%CI:[0.13,0.22]) was slightly more important than the dosing mode (RI:15.07%, 95%CI:[0.10,0.20). Although the production of inhibitors is the most affected side effect for hemophilia, patients would accept an additional 4.85% risk of inhibitors to reduce the annual bleeding rate from 12 times to 0 times, an additional 2.23% risk of inhibitors to change the dosing frequency from three times a week to once a week, and additional 1.97% risk from intravenous drip to subcutaneous.
Conclusions:
The annual bleeding rates and inhibitors risk are more highly valued by patients receiving prophylaxis. Physicians should take into account patients' preference in prophylactic treatment development and evaluation processes, so as to improve their treatment adherence and health outcome.
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