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

Abstract

Introduction: 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.

Methods: 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.

Results: 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).

Conclusion: 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.