Burden of Hereditary Angioedema in Adults: Impact of Disease and Patient Characteristics

Background: Hereditary angioedema (HAE) has a heterogeneous clinical presentation and symptoms can vary in severity and frequency. The aim of this post hoc analysis was to describe the burden of disease in patients with HAE according to impact of disease and patient characteristics. Methods: A cross-sectional, web-based survey in 13 countries was conducted to assess the burden of disease in patients aged ≥18 years with HAE. This post hoc analysis assessed disease control, HRQoL, anxiety, depression and work productivity by subgroups defined by sex, perception of disease control and HRQoL impairment. Patient-reported outcomes included the Angioedema Quality of Life (AE-QoL), Angioedema Control Test (AECT), Hospital Anxiety and Depression Scale (HADS), and Work Productivity and Activity Impairment: General Health (WPAI:GH) questionnaires. Descriptive statistics were used to describe the results by subgroups. Results: Of 260 respondents (189 female; 71 male) who completed the survey, 195/260 (75.0%) perceived their HAE was poorly controlled (AECT score <10) and 137/260 (52.7%) had moderate-to-large HRQoL impairment (AE-QoL score ≥39). Relative to male respondents, females reported higher HRQoL impairment (AE-QoL total score mean±SD 46.5±23.3 vs 33.3±20.1), a perception of more poorly controlled disease (AECT score 7.1±3.0 vs 8.3±3.0), and a higher proportion reported moderate or severe anxiety (33.9% vs 7.0%) and depression (12.2% vs 5.6%) according to the HADS. Respondents who perceived their HAE was poorly controlled had a higher number of HAE attacks in the last 6 months, reported greater HRQoL impairment, higher scores on HADS anxiety and depression subscales, and greater WPAI:GH work and activity impairment than those who perceived their HAE to be well controlled (AECT ≥10). Similarly, patients reporting moderate-to-large HRQoL impairment reported more HAE attacks in the last 6 months, higher scores on HADS anxiety and depression subscales, and greater WPAI:GH work and activity impairment than those with an AE-QoL score <39. Conclusion: In this post hoc analysis, the burden of HAE was consistently higher in females than males, in respondents who reported their disease to be poorly controlled versus well-controlled, and in those with moderate-to-large versus minimal HRQoL impairment. Findings support the use of patient-reported outcomes to assess burden of disease in routine clinical practice to understand the broader burden of disease beyond metrics such as attack rate, and to consider individual circumstances and experiences.