Hereditary angioedema (HAE) with C1-inhibitor deficiency is associated with painful, potentially fatal attacks affecting subcutaneous or submucosal tissues.
Objective
To evaluate HAE burden from the patients’ perspective.
Methods
This was a noninterventional survey of patients with HAE in the United States, conducted from March 17 to April 28, 2017. Patients were recruited through the US Hereditary Angioedema Association. Key eligibility criteria included the following: (1) aged 18 years and older, (2) self-reported physician diagnosis of HAE type I or II, (3) 1 or more HAE attacks or prodromal symptoms within the last year, and (4) receipt of HAE medication for an attack within the last 2 years. Descriptive analyses were conducted.
Results
A total of 445 patients completed the survey. Most patients (92.8%) were aged 18 to 64 years with HAE type I (78.4%) and had a positive family history (78.4%). Mean (SD) ages at symptom onset and diagnosis were 12.5 (9.1) and 20.1 (13.7) years, respectively. Most patients (78.7%) experienced an attack within the past month. The abdomen (58.0%) and extremities (46.1%) were commonly affected sites; pain (73.9%) and abdominal (57.0%) and nonabdominal (55.1%) swelling were frequently reported symptoms. Most patients (68.5%) had received or were currently receiving long-term prophylaxis. Most patients (88.8%) reported visiting allergists or immunologists, whereas 9.2% visited emergency departments or urgent care clinics. Per the Hospital Anxiety and Depression Scale, 49.9% and 24.0% of respondents had anxiety and depression, respectively. Mean Hereditary Angioedema-Quality of Life scores were generally lower with higher attack frequency. General health was “poor” or “fair” for 24.8% of patients. Mean (SD) percentage impairments were 5.9% (14.1%) for absenteeism, 23.0% (25.8%) for presenteeism, 25.4% (28.1%) for work productivity loss, and 31.8% (29.7%) for activity impairment.
Conclusion
Despite treatment advances, patients with HAE in the United States continue to have a high burden of illness.
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Ms. Long was affiliated with the US Hereditary Angioedema Association at the time of this analysis.
Disclosures: Dr Banerji reported receiving research funding from BioCryst and Shire, a Takeda company, and is on advisory boards for BioCryst, CSL Behring, Kalvista, Pharming, Pharvaris, and Shire. Ms. Davis is a part-time employee of RTI Health Solutions. Dr Brown, Ms. Hollis, and Ms. Hunter are full-time employees of RTI Health Solutions. Dr Jain and Dr Devercelli are full-time employees of Shire, a Takeda company, and hold stock or stock options in Takeda. Ms. Long has no conflicts of interest to disclose.
Funding sources: This research was funded by Shire Human Genetic Therapies, Inc, a Takeda company, Lexington, Massachusetts.