Development of a New Tool for Assessing Health-Related Quality of Life (QoL) in Patients with Hereditary Angioedema (HAE): The United States HAE Association (HAEA)-QoL
Saturday, March 3, 2018
South Hall A2 (Convention Center)
Sandra C. Christiansen, MD FAAAAI, Paula J. Busse, MD FAAAAI, Janette Birmingham, M.S., Aleena Banerji, MD, William R. Lumry, MD FAAAAI, Bruce L. Zuraw, MD
RATIONALE: Hereditary Angioedema (HAE) with C1-INH deficiency (HAE-C1-INH) presents with unpredictable episodes of angioedema, which can be potentially life-threatening, and is often disfiguring and interferes with patients’ daily activities. A tool to capture the health related quality of life (HRQoL) specific to HAE enables providers and patients to objectively measure the control of disease and the impact of new therapies for this rare disorder. To date, there has not been a questionnaire to measure HRQoL in patients with HAE based upon the management guidelines developed by the United States (US) Medical Advisory Board (MAB) of the HAE-Association (HAEA) and validated with US patients.

METHODS: A preliminary 41-item questionnaire was generated by physician members of the HAEA MAB, review of the literature and patient feedback. Content validity and item reduction were performed by administering the questionnaire to patients with HAE-C1-INH attending a US national HAE patient summit. Questions were removed by assessing response rates and exploratory factor analysis (EFA).

RESULTS: One hundred and sixty-eight patients with HAE-C1-INH completed the preliminary questionnaire (HAEA-QoL). Response rate and EFA assessment, stratified questions into 2 domains, “feelings” and “concerns,” and identified 12 questions for removal to create the next version of the HAEA-QoL.

CONCLUSIONS: A preliminary version of a HRQoL questionnaire for patients with HAE-C1-INH has been developed based upon US management guidelines for HAE and patient feedback. Validation studies needed for FDA approval of a patient reported outcome (PRO) to address reliability, criterion validity, and the ability to detect clinical change over time, are currently in progress.