ArticleAdvances in therapy2026
Estimating the Impact of Hemophilia A Treatment Administration on Patient Health Utility: Combining a Discrete Choice Experiment with Time Trade-Off Method.
Article in Advances in therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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8 authors.
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Abstract
introductionPreferences between treatment characteristics can be understood and quantified by combining discrete choice experiment (DCE) and time trade-off (TTO) (DCE
methodsA total of 119 PWHA from a hemophilia treatment center and the National Bleeding Disorders Foundation Community Voices in Research were recruited. Their sociodemographics, clinical characteristics, and EQ-5D-5L scores were gathered. Treatment attributes for the DCE survey were based on the core outcome set for hemophilia gene therapy (coreHEM). For DCE
resultsModerately burdensome treatment (24% treat > once/week) was reported by 67%. Mean EQ-5D-5L visual analogue score was 75; mean utility score was 0.684. All DCE attributes were significant; treatment administration was most important (2-3 intravenous [IV] infusions/week vs. a single IV infusion with 10-year durability, regression coefficient [RC], - 1.99), followed by mental health (always vs. no concern, RC, - 1.37), annual bleeding frequency (≥ 5 vs. none, RC, - 0.73), and chronic pain (yes vs. no, RC, - 0.36). For DCE
conclusionAll coreHEM outcomes are important for treatment choices of PWHA. One-time IV infusion with 5- to 10-year durability can provide important patient utility gains over lifelong repeated SQ injections or IV infusions.
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