A novel analysis of hemophilia treatment administration on patient utility
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Description
Objectives: Hemophilia A treatments differ in the method and frequency of administration. This study aims to elicit preferences of patients with hemophilia A (PWHA) and quantify the incremental impact of treatment attribute changes on health utility by utilizing a discrete choice experiment (DCE) and a DCE with time trade-off (TTO) DCETTO methodology. Methods: An analysis was performed of 115 PWHA (mean age 37 years, range 18-70) recruited from the Louisiana Center for Bleeding and Clotting Disorders at Tulane’s Hemophilia Treatment Center, and the National Hemophilia Foundation who participated in a web-based or in-clinic survey. Treatment attributes were based on the core outcome set for hemophilia gene therapy (coreHEM) and included method and frequency of administration, mental health, chronic pain, and annual bleeding rate. For the DCETTO, 10-, 15-, and 20-year durations were used. Patients completed 12 DCE and 12 DCETTO tasks. Choices were analyzed using conditional logistic models. And socio-demographic data, clinical characteristics and EQ-5D-5L were obtained from medical records or were self-reported. Results: Approximately 57% of PWHA reported moderately burdensome treatment (22% treat > once/week). The mean EQ-5D-5L Visual Analog Scale (VAS) was 75 and mean EQ-5D-5L utility score was 0.684. In the DCE all attributes were statistically significant, with administration being the most important attribute (2-3 times IV infusion per week vs. 10-year durability utility of -1.99), mental health (always concerned vs. no concern utility of -1.37), bleeding (5 or more vs. none utility of -0.73), and finally chronic pain (yes vs. no utility of -0.36). In the DCETTO, treatment with multiple IV infusions weekly was associated with an annualized utility decrement (0.046 vs. 10-year durability, 0.044 vs. 5-year). And treatment with multiple SQ injections monthly was associated with an annualized utility decrement (0.037 vs. 10-year durability, 0.030 vs. 5-year). Conclusion: All coreHEM outcomes are important for treatment choices of PWHA. A one-time IV treatment can provide important utility for PWHA over currently available treatments.