Impact of injectable HAE on-demand treatments on health-related quality of life: a patient and caregiver interview study

Background: Hereditary angioedema (HAE) attacks can be unpredictable, painful, and debilitating. Although studies described the burden of prophylactic HAE treatments on patient and caregiver health-related quality of life (HRQoL), few have explored the effects of injectable on-demand treatments on HRQoL. Objective: To understand the impact of injectable on-demand HAE treatments on HRQoL. Methods: Patients (aged ≥ 12 years) with ≥ 1 HAE attack in the prior 6 months and adult caregivers (aged ≥ 18 years) of patients of any age with HAE were recruited between July and October 2024 to complete a qualitative interview. Questions concerned on-demand injectable treatment use, impacts, and burden. Thematic analysis was used to identify key themes across responses. Results: The 25 study participants from the US and UK (17 patients; 8 caregivers), who completed the interview, highlighted emotional and logistical reasons for delaying or forgoing injectable on-demand treatment, including fear of needles, portability, and complexity of administration. All participants described at least one negative impact on HRQoL, including anxiety and pain associated with treatment administration, disruption of daily activities or work/school days, and impacts on personal relationships. Adolescent patients reported greater impacts than adult patients. Although indicated for self-administration, some adult and all adolescent patients reported needing assistance with administration of their injectable on-demand treatment. All participants expressed interest in an oral on-demand treatment for reasons including portability, pain-free administration, and ability to treat attacks earlier. Conclusion: This study highlights the unmet need for an on-demand treatment that allows for earlier, pain-free administration, ultimately increasing patient independence and improving HRQoL for both patients and caregivers.