Rethinking the management of hereditary angioedema

Background: Hereditary angioedema with C1INH deficiency (HAE-C1INH) is a rare, debilitating genetic disorder characterized by recurrent, unpredictable attacks. Although treatments exist, patients with HAE still alter their lives to avoid triggers and experience substantial physical, psychosocial, and financial burdens. Objective: To estimate the burden that HAE-C1INH patients experience despite currently approved therapies, aiming to identify unmet needs related to HAE, its therapies and the ability to achieve normalization of life. Methods: A web-based survey was conducted from March to April 2025 among 100 US adults with HAE-C1INH currently receiving long-term prophylaxis and/or on-demand therapies. Responses captured attack frequency, the impact of living with HAE, avoidance of attack triggers, and the patients’ unmet needs. Descriptive statistical analysis was conducted. Results: Even with treatment, 80% of respondents reported ≥1 HAE attack in the past year and 61% thought about HAE at least weekly. Mental health was the aspect that respondents felt was most impacted by HAE (54% of respondents), and 73% reported taking ≥2 measures to avoid attack triggers. Several concerns impacted the ability to reach normalization; lifetime use of medication was the most commonly reported concern (68% of respondents). The greatest unmet needs associated with long-term prophylaxis were cost- and access-related. Conclusion: Substantial unmet needs related to disease control and achieving normalization remain for patients with HAEC1INH, despite existing treatments. This study re-enforces the need to not only assess the frequency and severity of attacks, but also the psychosocial, mental, logistical, and financial burden of lifelong management of HAE-C1INH in clinical practice.