Barriers to Normalization Persist for U.S. Patients With Hereditary Angioedema
Despite advances in long-term prophylaxis (LTP), many people living with hereditary angioedema (HAE) in the United States continue to fall short of “normalization,” a key treatment goal outlined in clinical guidelines, according to results of a patient survey recently published.
Study Findings
The cross-sectional, web-based survey included 100 U.S. adults living with HAE who were receiving on-demand (OD) therapy, LTP, or both. Most respondents (89%) reported use of LTP, while only 11% relied solely on OD therapy.
Clinical control remained suboptimal. Over the prior year, just 20% of respondents were attack-free, and more than one-third (34%) experienced at least one attack per month. Patients’ emotional burden was substantial; more than half (51%) of participants reported anxiety and frustration during attacks.
Cognitive and psychological impact was also evident. Nearly two-thirds of respondents (61%) reported thinking about HAE at least weekly, and one-third (33%) thought about the disease every day over the past year. When asked to rate how “normalized” they felt on current therapy using a 10-point scale, the mean score was 7.0 (SD, 2.4), suggesting moderate but incomplete normalization.
Concerns about chronic therapy emerged as a dominant theme. Lifetime medication use was the highest-rated barrier to normalization, with 68% rating it as a significant concern. When asked how current LTPs could be improved to enhance normalization, 55% ranked “no lifetime medication” among their top 3 priorities, and 30% ranked it as the single most important factor. Many respondents indicated they would feel “relieved,” “ecstatic,” or “grateful” if their HAE could be controlled without ongoing medication.
Clinical Implications
For allergists and immunologists managing HAE, these findings underscore that biochemical control does not necessarily equate to patient-perceived normalization. Persistent attacks, ongoing vigilance, and treatment fatigue may continue even among patients receiving LTP.
The high prevalence of anxiety, frequent disease-related thoughts, and concern about lifelong therapy highlight the importance of incorporating patient-reported outcomes into routine care. Clinicians should regularly assess not only attack frequency but also emotional burden, treatment satisfaction, and expectations around long-term disease management.
These results also have implications for shared decision-making. As newer therapeutic strategies and investigational approaches aim to reduce treatment burden or duration, understanding patient priorities—particularly the desire to avoid lifelong medication—may help guide individualized treatment planning and future innovation in HAE care.
Expert Commentary
According to the study authors, the findings suggest that chronic management strategies themselves may limit patients’ ability to feel fully “normalized.” The authors emphasize that even with modern therapies, many U.S. patients continue to experience attacks and ongoing psychological burden, indicating an unmet need beyond attack prevention.
Persistent attacks and concern about lifelong medication use point to the need for therapies and care models that address both clinical and humanistic outcomes.
Reference
Busse P, Wilson K, Farkas H, et al. Barriers to achieving normalization among people living with HAE on existing treatments. Ann Allergy Asthma Immunol. 2025;135(5 Suppl):S38-S39.
