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.