Introduction
Preferences between treatment characteristics can be understood and quantified by combining discrete choice experiment (DCE) and time trade-off (TTO) (DCETTO) methods. The goal is to elicit preferences for people with hemophilia A (PWHA) and quantify impacts of treatment attribute changes on patient health utility with DCETTO.
Methods
A 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 DCETTO, 10-, 15-, and 20-year life durations were used. In total 115 PWHA completed 12 DCE and DCETTO tasks, which were analyzed using conditional logistic models.
Results
Moderately 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 DCETTO, multiple IV infusions weekly and multiple monthly subcutaneous (SQ) injections were associated with annualized utility decrements (0.046 vs. 10-year durability, 0.044 vs. 5-year; 0.037 vs. 10-year, 0.030 vs. 5-year, respectively).
Conclusion
All 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.
Melody S Benton, Dongzhe Hong, Mark W Skinner, Louis P Garrison, Milad Karimi, Er Chen, Henry Mead, Lizheng Shi
Disease Category: Blood disorders
Disease Name: Haemophilia/hemophilia and other bleeding disorders
Age Range: 18 - 120
Sex: Male
Nature of Intervention: Gene therapy
- Consumers (patients)
- Patient perspectives
- Discrete Choice Experiments
- Survey