Health-related quality of life among children with hereditary angioedema

作者：Engel Yeger Batya; Farkas Henriette; Kivity Shmuel; Veszeli Nora; Kohalmi Kinga Viktoria; Kessel Aharon^*

来源：Pediatric Allergy and Immunology, 2017, 28(4): 370-376.

DOI：10.1111/pai.12712

• C1-inhibitor
• children
• complement-1 esterase inhibitor defects
• hereditary angioedema
• PedsQL (TM) 4.0
• quality of life
• symptom assessment

Background: The clinical expressions of hereditary angioedema with C1-inhibitor deficiency (C1-INH-HAE) and its related burden may negatively affect patient quality of life. This study aimed to assess health-related quality of life (HRQoL) in children with C1-INH-HAE.
Methods: Children (N = 98: 34 C1-INH-HAE patients, 64 healthy controls) aged 3-18 years were recruited in Israel and Hungary. All individuals completed a demographic questionnaire, a disease activity and site questionnaire, and the Pediatric Quality of Life Inventory (PedsQL (TM)) 4.0 Generic Core Scales (child self-report and maternal proxy report) to assess HRQoL.
Results: Among C1-INH-HAE patients, nine (26.5%) had 1-5 attacks/year, six (17.6%) had 6-18 attacks/year, eight (23.5%) had 25-60 attacks/year, and 11 (32.4%) were asymptomatic over the previous year. Children with C1-INH-HAE attacks demonstrated lower HRQoL than healthy control children across the total score, school, and psychosocial dimensions of the PedsQL (TM). The number of C1-INH-HAE attacks negatively correlated with the total HRQoL score (r = -0.48, p = 0.008), school-related HRQoL (r = -0.39, p = 0.02), and psychosocial HRQoL (r = -0.43, p = 0.01). Patients with multisite laryngeal, abdominal, and peripheral C1-INH-HAE attacks had a lower HRQoL compared with those who experienced solely peripheral attacks across the total score (p = 0.04), physical (p = 0.04), and school (p = 0.02) domains. There was no significant difference between asymptomatic C1-INH-HAE patients and healthy controls.
Conclusions: Children with symptomatic C1-INH-HAE demonstrate impaired HRQoL compared with healthy controls. HRQoL was affected by the frequency and site of C1-INH-HAE attacks and mostly in the school and physical domains.

• 出版日期2017-6