The hereditary angioedema burden of illness study in Europe (HAE-BOIS-Europe): background and methodology

Abstract

BackgroundHereditary angioedema (HAE) is a rare but serious disease marked by swelling attacks in the extremities, face, trunk, airway, or abdominal areas that can be spontaneous or the result of trauma and other triggers. It can be life-threatening due to the risk of asphyxiation. While there have been major advancements in our understanding of the immunogenetics of HAE, there are significant gaps in the literature regarding understanding of the humanistic and economic impact of the disease, particularly in Europe. The purpose of the HAE Burden of Illness Study-Europe (HAE-BOIS-Europe), the development and methodology of which is described here, is to better understand the management and impact of HAE from the patient perspective in Europe.Methods/DesignThis is a cross-sectional study in which retrospective data were also collected being conducted in Denmark, Germany and Spain. The study is open to patients ages 12 and older with a diagnosis of HAE-I or HAE-II. Data collection includes: (i) a survey on individuals’ health care resource use, direct and indirect medical costs, impact on work and school, treatment satisfaction, and emotional functioning (via the Hospital Anxiety and Depression Scale); and (ii) one-on-one interviews to collect detailed descriptive data and patient testimonials on the impact of HAE on patients’ health-related quality of life.DiscussionThe present manuscript describes the development and plans for implementing a multi-country European study with the aim of characterizing the humanistic and economic burden of HAE from the patient perspective. This study will help raise awareness of HAE as a rare but debilitating condition with wide-ranging impacts.

Safety of C1-Esterase Inhibitor in Acute and Prophylactic Therapy of Hereditary Angioedema: Findings from the Ongoing International Berinert Patient Registry

“…As a chronic disease with the potential for high morbidity and mortality, HAE has a substantial quality-oflife impact on affected patients and their families. [2][3][4][5][6][7][8][9][10] Although management strategies are individualized, all patients with HAE should have easy access to an on-demand therapy for treatment of acute attacks and education regarding short-term prophylaxis before known triggers of HAE, including invasive medical or dental procedures. In addition, some patients may require longterm prophylaxis to prevent HAE attacks.…”

Challenges of C1-Inhibitor Concentrate Self-Administration

“…Awareness of hereditary angioedema (HAE) is extremely low and diagnosis of the condition may take several years with a mean delay to diagnosis of as much as 16 years [1]. This emphasises the urgent need to raise awareness of the disease among clinicians and within families who carry a hereditary predisposition to the condition.…”

Current Medical Management of Hereditary Angioedema: Follow-up Survey of US Physicians

“…1,2 The physical distress and lifestyle interruptions associated with HAE have a substantial impact on quality of life for both patients and their families. [7][8][9][10][11][12][13][14][15] The general goals of HAE management include minimizing the overall morbidity of the condition and preventing fatalities. Specific strategies include long-term prophylactic drug administration, short-term prophylaxis before potential triggers, and on-demand treatment of attacks when they occur.…”