Patient Experience with Treatment of HAE Attacks: Results from a Real-World Survey of Physicians and Their Patients

Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent, unpredictable, and potentially life-threatening angioedema attacks. Few reports describing physician and patient perspectives on their experiences with HAE, long-term prophylaxis (LTP), and on-demand treatment regimens exist. This analysis aimed to address remaining critical gaps, particularly regarding patient attitudes toward treatment adherence, symptom experience, and timing to relief onset, by capturing physician and patient insights on these topics. Data are from the Adelphi Real World HAE Disease Specific Programme™, a cross-sectional, retrospective survey of physicians and their patients conducted across Europe, Japan, and the US from January 2023 through January 2024. Surveyed physicians were directly involved in the management of at least two patients with HAE per month. Eligible patients had experienced at least one attack since diagnosis. Physicians completed an online Patient Record Form (PRF) based on data from patients’ medical charts and their clinical judgment. Each patient with a physician-completed form was invited to complete a voluntary self-reported questionnaire. Participating physicians (n = 225) completed PRFs for 1131 patients (age range: 3–82 years); 279 patients completed the questionnaire. Most patients were female (physician reported: 607/1131 [54%]; patient reported: 145/279 [52%]). Overall, 48% of patients in the physician-reported groups and 51% in the patient-reported group experienced one to two attacks during the 12 months before the survey. The top reason for not treating the most recent attack was perceiving it as mild/not severe or limiting (physician reported: 54%; patient reported: 59%). The top reason for suboptimal adherence to LTP in the physician- and patient-reported groups was forgetting to take medication (45% and 51%, respectively). This analysis increases our understanding of the lived experience of HAE and helps address the remaining gaps in treatment satisfaction and adherence within the HAE community. Hereditary angioedema (HAE) is a rare condition causing unexpected swelling attacks that can be painful and sometimes life‑threatening. Effective medications are available to prevent or relieve attacks, but there is little information about how people with HAE use these treatments day to day. This study looked at how often HAE attacks occur, the symptoms associated with attacks, how often people treat attacks, how people like to treat attacks, and how HAE affects daily activities. Information was collected from the point of view of people with HAE and their doctor. Treating doctors completed a survey between January 2023 and January 2024. Their practices were in Europe, Japan, and the US. Doctors shared information on people with HAE based on their experiences and people’s anonymized medical records. The same people with HAE could also share their views of HAE and their treatment habits. Overall, 225 doctors provided information for 1131 people with HAE; 279 of these people shared their views. About half of people with HAE who completed the survey had one or two attacks in the year before. In all, 135 people according to surveyed doctors and 30 surveyed individuals with HAE felt their most recent attack was mild and decided not to take any medication for it. Forgetting to take medication was the most common reason for not using treatment that can make attacks happen less often. Improving awareness of ongoing challenges with treatment satisfaction and adherence may help healthcare providers better support people living with HAE.