Achieving a normal life in hereditary angioedema: Quality of life and treatment gaps among German HAE patients.

Abstract

Background: Hereditary angioedema (HAE) is a rare genetic disease characterized by recurrent swelling attacks. Current guidelines for HAE management emphasize achieving complete disease control to normalize patients' lives. Quality of life (QoL) differences between patients with 0 attacks and those with persistent attacks remain to be explored.

Materials and methods: The German patient organization for individuals affected by hereditary angioedema, HAE Vereinigung e.V. conducted an online survey with 122 HAE patients in Germany in 2024. Participants were categorized according to their therapy - long-term prophylaxis (LTP) or on-demand therapy (ODT) - and according to their attack frequency over the last 6 months. Patient-reported outcomes for functional, emotional, and social impacts were analyzed to evaluate QoL.

Results: Although 83% of patients expressed satisfaction with their treatment, 59% of patients still had attacks. Patients on LTP reported significantly fewer attacks (p < 0.001) and higher QoL compared to those on ODT (p < 0.001). Patients with 0 attacks consistently showed significantly better outcomes across all QoL domains than those with 1 or more attacks (p < 0.001).

Conclusion: The findings highlight that even minimal residual disease activity can meaningfully reduce QoL. Achieving complete attack freedom, rather than partial control, is necessary to restore normalcy for patients with HAE. Hence, regular adjustments of the HAE management plans based on patient-reported outcomes are crucial to ensure that treatment strategies address both medical and QoL needs.