- D1.336 - Barriers to Normalization with Existing Treatments Among People Living with Hereditary Angioedema in Europe

Even with many available treatments, people living with hereditary angioedema (HAE) experience multi-faceted burdens that impact their ability to feel “normalized”, which is a treatment goal per HAE clinical guidelines. This study evaluates the burden of HAE and its treatments, including humanistic burden and aspects of normalization experience among patients with HAE in three European countries.

A cross-sectional survey was conducted among patients with Type I and II HAE in Germany (DE), France (FR), and the United Kingdom (UK).  Data were summarized using descriptive statistics.

Overall, 87 respondents across DE, FR, and the UK participated; the percentage of respondents that used on-demand treatment (ODT) ranged from 45%-52% and the percentage that used long-term prophylaxis (LTP) with or without ODT ranged from 48%-55%. In the past year, only 2% were attack-free and 21% experienced ≥3 attacks per month. A total of 68% had thoughts about their HAE at least weekly. Of those who experienced attacks (n=85), 71% impacted the face and 25% had a laryngeal attack in the past year.

When asked, 87% indicated at least one potential trigger they actively avoid. The specific measures taken to avoid attack triggers varied among respondents: physical (91%), emotional (71%), other (53%), professional/educational decisions (51%), social (43%), and clinical (18%).  On average, respondents reported missing ~10 days of school (43/87) or work (59/87) in the past 6 months. The top aspects of normalization that continued to be a significant concern (rated ≥5; 7-point scale) in ≥60% of respondents were related to travel, HAE attacks, and chronic medication use. Only 10% of respondents rated themselves completely normalized and 67% as highly normalized (rated 10 and ≥8 respectively; 10-point scale). The top, high unmet needs of current LTPs (rated ≥5; 7-point scale) varied between the three countries; overall, all ten unmet needs presented were rated as high unmet needs by ≥45% respondents.

Patients with HAE in DE, FR, and the UK on treatments still experience significant burden of HAE and use of lifelong treatments; however, they perceive themselves as having a high level of normalization, which highlights a disconnect in treatment expectations. With more than half of respondents reporting continued attacks and considerable unmet needs while on treatment, broader aspects need to be considered in evaluating patients achieving “normalization” with HAE treatments.