Validated Tool Offers Structured Measure of Hemophilia Patients’ Device Preferences

As treatment options for hemophilia evolve to include a range of administration methods, understanding how patients and caregivers perceive different injection devices is increasingly critical. A recent study evaluated the Hemophilia Device Handling and Preference Assessment (HDHPA) questionnaire, a new instrument designed to capture patient comprehension, relevance, and comprehensiveness in evaluating pen-injectors.

The study aimed to confirm whether the HDHPA is a valid tool for assessing how individuals with hemophilia and their caregivers view device handling and preferences. By establishing content validity through direct patient input, researchers sought to ensure the tool could support studies comparing pen-injectors with traditional devices. In the context of payer and managed care decision-making, validated instruments like the HDHPA can strengthen evidence supporting patient adherence, satisfaction, and long-term outcomes.

Conducted in the US, this cross-sectional study recruited participants from an ongoing pen-injector handling and preference trial. All participants had previously completed the 10-item HDHPA questionnaire before engaging in semi-structured cognitive interviews. The interviews explored the clarity of instructions, comprehension of individual items, and the appropriateness of response options. Importantly, this approach emphasized whether the questionnaire was both accessible to patients and sufficiently comprehensive to reflect their experience with hemophilia treatment devices.

The study included 20 participants, representing a balanced cross-section of the hemophilia community. Half were adult patients, while the remainder comprised adolescents (25%) and caregivers (25%). Reflecting hemophilia’s known demographics, 75% of participants were male. This distribution allowed for assessment of the tool across both self-administrators and those involved in treatment support.

Results demonstrated a high degree of comprehension and relevance across all aspects of the HDHPA. Between 89% and 100% of participants understood the instructions provided, while 94% to 100% clearly comprehended the 10 questionnaire items. Furthermore, all 20 participants reported that the instrument was relevant and comprehensive in evaluating the pen-injector device and in comparing it to their current treatment system.

The validation of the HDHPA has implications that extend beyond clinical research. For payers and managed care organizations, tools that systematically capture patient preferences provide valuable insights into adherence potential. Selecting devices that align with patient or caregiver preferences may translate into better treatment consistency, improved outcomes, and potentially lower overall costs associated with complications of poor adherence.

By confirming content validity, the study positions the HDHPA as the first structured instrument specifically tailored to hemophilia patients’ experiences with injection systems. This addresses a previously unmet need in both clinical practice and outcomes research. Moreover, it underscores the growing importance of incorporating patient-reported perspectives into device and therapy evaluations.

The findings demonstrate that the HDHPA questionnaire is both comprehensible and relevant for adult and adolescent hemophilia patients as well as caregivers. It offers a standardized approach to capturing device handling preferences.

Reference

Rasmussen NK, Shah S, Tsai J, et al. Content validity of the hemophilia device handling and preference assessment questionnaire - a cognitive debrief study in patients with hemophilia and their caregivers. Patient Prefer Adherence. 2025;9:19:2807-2816. doi:10.2147/PPA.S529134