PB0654 - Burden of Treatment for Adult and Adolescent People with Haemophilia (PwH): Concept Elicitation Results Supporting the Development of the Hemophilia Treatment Experience Measure (Hemo-TEM)

Background: Currently available treatments for PwH have resulted in significant improvements in quality of life. However, PwH may still experience considerable treatment burden due to technical, physical, logistical, and emotional aspects of self-administering intravenous infusions. Most research to date has focused on disease burden rather than treatment burden.

Aims: This study’s purpose was to investigate treatment burden among adult and adolescent PwH and collect data to support the content validity of a new disease-specific patient-reported outcome measure of treatment burden.

Methods: Following U.S. Food and Drug Administration guidelines, interviews were conducted with 30 adult males with moderate or severe haemophilia, with or without inhibitors, and who self-infused prophylactically or on demand. Interviews were analysed and coded using an adapted grounded theory approach to determine overarching themes and concepts. A theoretical model of treatment burden was then developed, and items for the new Hemo-TEM measure were generated and cognitively debriefed in a total of 35 PwH (15 adults and 20 adolescents).

Results: Qualitative analysis revealed that saturation of concepts was reached with 5 treatment burden domains: injection difficulties; adherence; emotional burden; physical burden; and interference with daily life. Sub-concepts endorsed by patients included inserting the needle correctly (53%) and fitting treatment into daily schedule (43%) (injection difficulties); missed (53%) and delayed (40%) treatments (adherence); worry (47%) and anxiety/nervousness (27%) (emotional burden); blown veins (50%) and pain (50%) (physical burden); and travel (73%) and work (40%) (interference with daily life) (Table 1). The cognitive debriefing resulted in a draft measure of 30 items, which were found to be understandable and relevant.

Conclusion(s): The content validity of the Hemo-TEM is supported by these qualitative findings. The validation-ready Hemo-TEM version contained items on treatment difficulties, adherence, emotional burden, physical burden, and interference with daily life and was found acceptable by adult and adolescent patients.