Hereditary Angioedema (HAE)
Conditions
Keywords
survey, on-demand treatment, hereditary angioedema, burden, caregivers, quality of life
Brief summary
The study has been designed as a French, multicentric survey study to obtain data to describe the burden of on-demand treatment for patients with hereditary angioedema (HAE). The online survey assesses various aspects of HAE and in particular the burden of HAE from the patients' and caregivers' perspectives.
Detailed description
The study has been designed as a French, multicentric survey study to obtain data to describe the burden of on-demand treatment for patients with hereditary angioedema (HAE). The study will also explore the burden related to the administration of intravenous or subcutaneous on-demand treatments. The study was designed to collect data concerning the burden of HAE in patients with HAE (during HAE attacks and between attacks) and in caregivers. The online survey will collect data to describe the characteristics of patients with HAE, the disease characteristics, details concern various aspects of HAE attacks, and the burden of HAE in patients and their caregivers.
Interventions
None listed
Sponsors
University Hospital, Grenoble
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
For the patient population: 1. Patients with a confirmed diagnosis of HAE with a deficit in the C1 inhibitor. 2. Aged ≥12 years 3. Having consulted for HAE in the last 3 months and with a medical file. For the caregiver population: 1\. A person identified by the patient as part of their support group and who provides support with the management of the patients HAE (including family members, friends, spouse, etc.)
Exclusion criteria
For the patient population: 1. Opposes to participating in the study. 2. Patients is under guardianship or deprived of their liberty.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To describe the burden associated with the on-demand treatment of HAE attacks | The survey will focus on HAE attacks that have occurred within the last 12 months. | The burden associated with the on-demand treatment of HAE attacks from the patient's and caregiver's perspective, will be described in terms of: * The frequency of HAE attacks. * The severity of HAE attacks. * Disease control. * Events that trigger the HAE attacks. * Frequency of injections for treating HAE attacks. * Delay in injections for treating HAE attacks. * Hospitalization for HAE attacks. * Impact on quality of life (during HAE attacks). * Impact on quality of life (between HAE attacks). * Social impact. * Financial impact. * Burden of the caregivers. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Use of on-demand treatments for HAE attacks | The survey will focus on HAE attacks that have occurred within the last 12 months. | The survey will collect data to for the following: * To estimate the number of patients that delay or do not use injectable on-demand treatments for HAE. * To identify the reasons why patients, delay or do not use injectable on-demand treatments for HAE attacks. * To study the potential benefits of introducing an oral on-demand treatment for HAE attacks. |
Countries
France
Outcome results
None listed