Quantifying the burden of disease and perceived health state in patients with hereditary angioedema in Sweden

Abstract

Hereditary angioedema (HAE) due to C1 inhibitor deficiency is a rare disease characterized by attacks of edema, known to impact quality of life (QoL). This study investigates the burden of HAE in Swedish patients, both children and adults. We used a retrospective registry study of Swedish patients with HAE, captured by the Sweha-Reg census. Data were collected using a paper-based survey. Patients completed EuroQoL 5 Dimensions 5 Levels (EQ5D-5L) questionnaires for both the attack-free state (EQ5D today), and the last HAE attack (EQ5D attack). Questions related to patient's age and sex and other variables, such as attack location and severity, were included to better understand the burden of HAE. EQ5D-5L values were estimated for the two HAE disease states. Patient-reported sick leave was also analyzed. A total of 103 responses were analyzed from 139 surveys (74% response rate). One hundred one reported an EQ5D today score (mean, 0.825) and 78 reported an EQ5D attack score (mean, 0.512) with significant differences between the two states (p < 0.0001). This difference was observed for both mild (p < 0.05), moderate (p < 0.0001), and severe attacks (p < 0.0001). Attack frequency had a negative effect on EQ5D today. Patients with >30 attacks a year had a significantly lower EQ5D today score than those with less frequent attacks. Of 74 participants, 33 (44.6%) had been absent from work or school during the latest attack and, of those with a severe attack, 81% had been absent. HAE has a significant impact on QoL both during and between attacks and on absenteeism during attacks.

The asthma‐chronic obstructive pulmonary disease overlap syndrome and its challenge for the allergist‐immunologist

“…Despite the well-established burden of illness associated with hereditary angioedema (HAE) 17,18 and the fact that symptoms often appear early in life and accelerate around puberty, there is a paucity of both data and treatment options for HAE in children in the United States. Bennett and Craig 19 provide insight into the unmet need by summarizing the published data and performing a retrospective chart review on 25 children with HAE.…”

Disorders for which the allergist-immunologist is particularly well-qualified to treat

“…Previous issues of the Proceedings have addressed unmet needs and treatment advances in hereditary angioedema (HAE). [11][12][13][14][15][16][17][18][19][20][21][22] Within the pages of the current issue appear four articles which provide new information on the familial occurrence, differential diagnosis and treatment of HAE. Despite the known predominant autosomal dominant inheritance of HAE, diagnostic testing remains underutilized in those potentially susceptible but currently undetected patient populations.…”

United airway disease

“…[7][8][9][10][11][12][13][14][15][16] Directly relating to these issues, three articles appear within the pages of this issue that focus on this heterogeneous disease. [17][18][19] In the first article, Felder et al, 17 investigate the emergency department presentation patterns of undifferentiated angioedema at two tertiary care hospitals over a three year period.…”