title: Health-related quality of life among children with hereditary angioedema.
abstract: 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.
  
  
  
  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™) 4.0 Generic Core Scales (child self-report and maternal proxy report) to assess HRQoL.
  
  
  
  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™. 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.
  
  
  
  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.
authors.affiliation: Department Faculty of Social Welfare and Health Sciences, University of Haifa Occupational Therapy, Haifa, Israel.
authors.forename: Batya
authors.initials: B
authors.lastname: Engel-Yeger
authors.affiliation: 3rd Department of Internal Medicine, Hungarian Angioedema Center, Semmelweis University, Budapest, Hungary.
authors.forename: Henriette
authors.initials: H
authors.lastname: Farkas
authors.affiliation: Allergy Department, Tel-Aviv Sourasky Medical Center, Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel.
authors.forename: Shmuel
authors.initials: S
authors.lastname: Kivity
authors.affiliation: 3rd Department of Internal Medicine, Hungarian Angioedema Center, Semmelweis University, Budapest, Hungary.
authors.forename: Nóra
authors.initials: N
authors.lastname: Veszeli
authors.affiliation: 3rd Department of Internal Medicine, Hungarian Angioedema Center, Semmelweis University, Budapest, Hungary.
authors.forename: Kinga Viktória
authors.initials: KV
authors.lastname: Kőhalmi
authors.affiliation: Division of Allergy and Clinical Immunology, Technion Faculty of Medicine, Bnai Zion Medical Center, Haifa, Israel.
authors.forename: Aharon
authors.identifier: 0000-0003-4141-2100
authors.initials: A
authors.lastname: Kessel
mesh_terms: D000293:Adolescent; D054179:Angioedemas, Hereditary / Q000453:epidemiology* / Q000235:genetics; D002648:Child; D002675:Child, Preschool; D050718:Complement C1 Inhibitor Protein / Q000235:genetics; D018450:Disease Progression; D005260:Female; D006801:Humans; D006814:Hungary / Q000453:epidemiology; D007557:Israel / Q000453:epidemiology; D008297:Male; D011788:Quality of Life; D012574:Schools; D011795:Surveys and Questionnaires
chemical_list: D050718:Complement C1 Inhibitor Protein
keywords: C1-inhibitor; PedsQL™ 4.0; children; complement-1 esterase inhibitor defects; hereditary angioedema; quality of life; symptom assessment