A Burden of Illness Chart Review Study in Hereditary Angioedema
Research type
Research Study
Full title
A Retrospective Observational Chart Review Study Evaluating the Burden of Illness and Treatment Patterns in Hereditary Angioedema Type I and II
IRAS ID
312025
Contact name
Ania Manson
Contact email
Sponsor organisation
Takeda
Duration of Study in the UK
1 years, 0 months, 0 days
Research summary
Research Summary:
This is an observational, multi-country, international, retrospective cohort study consisting of a medical chart review of patients 12 years of age and over with inadequately controlled hereditary angioedema (HAE) type I or type II.
Although various short-term and long-term options are available to prevent hereditary angioedema (HAE) attacks, the disease may still be inadequately controlled. There is a need, therefore, to assess and understand the burden of the disease as well as real-world treatment patterns of current HAE-specific therapies in order to identify the need for new therapies.
Approximately 210 participants at 30 centers in at least 15 European and Middle Eastern countries (with possible expansion to Canada), with a two sites in UK will be enrolled on the study.De-identified data (i.e., anonymous to non-site staff) on patient demographics, HAE medical history and information on diagnostics, treatments and disease course assessments that are routinely performed in accordance with current guidelines and/or local standard of care will be collected from patient medical records and entered into an EDC (electronic data capture) system over multiple data collection waves.
In addition, patient outcomes on HRQoL (Health-related quality of life) will be collected remotely and electronically. These HRQoL assessments include the Angioedema Quality of Life (AE-QoL; patients ≥18 years old), EQ-5D-5L (patients ≥16 years old) and EQ-5D-Y (patients 12–15 years old).
This study is observational and primarily retrospective; therefore, information pertaining to patient care will already be documented in patient medical records at the time of chart abstraction. This study does not result in interference with standard medical care; thus, it will not impact the treatment of study patients.
Lay summary of study results: Not available.REC name
Yorkshire & The Humber - South Yorkshire Research Ethics Committee
REC reference
22/YH/0133
Date of REC Opinion
9 Jun 2022
REC opinion
Favourable Opinion