RESUMO
BACKGROUND: Hereditary angioedema (HAE) attacks are unpredictable, cause a substantial and enduring burden of illness, and are potentially fatal. Because of issues unique to the US health care system, there is a need for a US-validated, HAE-specific quality of life (QoL) instrument. OBJECTIVE: To develop and validate a US HAE-specific QoL instrument according to US Food and Drug Administration guidelines and established methodologies. METHODS: We generated 41 QoL-related items likely relevant to US patients with HAE due to C1 inhibitor (C1INH) deficiency (HAE-C1INH) and performed a 10-patient pilot study to refine the question wording. A total of 415 US patients with HAE-C1INH completed the initial 41-item instrument online, thereby providing data for item reduction, factor analysis, and the assessment of validity and reliability. We used a multiple linear regression to identify the drivers of the total and domain scores. Convergent validity analysis was used to assess the extent to which the HAE-C1INH QoL instrument (HAE-C1INH-QoL) is theoretically related to the angioedema-QoL instrument (AE-QoL). RESULTS: Item reduction and factor analysis yielded a final instrument of 31 items across 5 domains, and the assessment analysis showed that the HAE-C1INH-QoL is valid and reliable. Attack frequency and severity were statistically significant factors that influenced the total and domain scores. Correlation analysis of the 2 instruments indicated that 8 items of the HAE-C1INH-QoL were not included or well-described in the AE-QoL. CONCLUSION: The HAE-C1INH-QoL is the first HAE-specific QoL tool validated in the United States. When compared with the AE-QoL, the items in our instrument are more relevant to US patients with HAE.
RESUMO
BACKGROUND: Hereditary angioedema (HAE) is a rare and potentially fatal genetic disease associated with recurrent and unpredictable episodes of angioedema. Although modern therapies have dramatically increased quality of life, insurance changes, delays, and denials are becoming more common. OBJECTIVE: To examine the impact of insurance delays and denials on patient health and well-being. METHODS: A total of 20 patients with HAE (type 1 and 2) who recently experienced insurance delays or denials completed an online survey, and 19 participated in a follow-up focus group. The survey and focus group addressed the impact of insurance challenges on the use of health care services, work/school attendance, and anxiety. Three independent reviewers coded each focus group transcript using a thematic saturation approach. RESULTS: A total of 70% of participants reported an increased frequency of angioedema attacks resulting from insurance delays or denials. More than 50% missed work/school days because of increased attacks, and 90% reported greater anxiety. Twenty-five percent of respondents reported more urgent care or emergency department visits. In focus groups, participants identified specific ways that losing access to medication had a negative impact on their health, family, and work/school life. Insufficient notification of health insurance policy changes and the time and effort required to regain access to medications compounded patients' frustration and anxiety. CONCLUSION: Insurance delays and denials have significant impacts on individuals with HAE including (1) increased urgent care and emergency department visits, (2) missed work/school days, (3) higher levels of anxiety, and (4) a negative impact on family life.