Investigation of the Impact of Hereditary Angioedema (HAE) on Health-Related Quality of Life: A Systematic Literature Review
Author(s)
Charlotte Heeks, BSc, MSc1, Madeleine Thursfield, MA1, Marie Prett, BSc, MSc2, Laura Everett, BSc, MSc2, Alice Laglia, BSc, MSc2, Brian Kwizera, MBiochem2.
1Otsuka Pharmaceutical Europe Ltd, Windsor, United Kingdom, 2FIECON, a Herspiegel company, London, United Kingdom.
1Otsuka Pharmaceutical Europe Ltd, Windsor, United Kingdom, 2FIECON, a Herspiegel company, London, United Kingdom.
OBJECTIVES: HAE is a rare genetic disorder characterized by debilitating, unpredictable swelling attacks, commonly affecting the extremities, stomach, and throat. This review examined the existing health-related quality of life (HRQoL) studies for patients with HAE.
METHODS: A systematic literature review (SLR) was conducted in June 2024 to identify HRQoL studies published in English in individuals with Type I and Type II HAE. The databases searched included Embase, Centre for Reviews and Dissemination Health technology assessment (CRD HTA), European Quality of Life (EuroQoL), and the ScHARR Health Utilities Database (ScHARRHUD). The SLR was conducted in line with Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) guidelines.
RESULTS: Overall, 5,987 references were screened, with 197 HRQoL-related references considered for secondary screening. From 100 HRQoL references accepted, 31 were full-text journal articles reporting 27 unique studies that assessed treatment with berotralstat (n=4), C1-INH (n=3), donidalorsen (n=3), garadacimab (n=2), avoralstat, danazol, and lanadelumab (n=1 each); ten included a placebo comparator and twelve were non-interventional studies. The HAE-specific Angioedema Quality of Life (AE-QoL) questionnaire was used in 17 of these studies, while EuroQoL 5-dimension (EQ-5D), the preferred measure of HRQoL for many health technology assessments, was reported in four studies. Evidence on HRQoL impact during patients’ HAE attacks was limited, with only one study, a non-interventional, cross-sectional survey, comparing HRQoL during and in-between attacks. High AE-QoL scores at initial assessment and in placebo arms demonstrate the poor HRQoL among patients with HAE.
CONCLUSIONS: Only one study was identified that captured HRQoL during and between patients’ HAE attacks. There remains difficulty in accurately quantifying the impact of each attack on HRQoL, likely due to the transient and unpredictable nature of attacks. This highlights the need for further research to improve understanding of how to measure patient HRQoL during attacks.
METHODS: A systematic literature review (SLR) was conducted in June 2024 to identify HRQoL studies published in English in individuals with Type I and Type II HAE. The databases searched included Embase, Centre for Reviews and Dissemination Health technology assessment (CRD HTA), European Quality of Life (EuroQoL), and the ScHARR Health Utilities Database (ScHARRHUD). The SLR was conducted in line with Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) guidelines.
RESULTS: Overall, 5,987 references were screened, with 197 HRQoL-related references considered for secondary screening. From 100 HRQoL references accepted, 31 were full-text journal articles reporting 27 unique studies that assessed treatment with berotralstat (n=4), C1-INH (n=3), donidalorsen (n=3), garadacimab (n=2), avoralstat, danazol, and lanadelumab (n=1 each); ten included a placebo comparator and twelve were non-interventional studies. The HAE-specific Angioedema Quality of Life (AE-QoL) questionnaire was used in 17 of these studies, while EuroQoL 5-dimension (EQ-5D), the preferred measure of HRQoL for many health technology assessments, was reported in four studies. Evidence on HRQoL impact during patients’ HAE attacks was limited, with only one study, a non-interventional, cross-sectional survey, comparing HRQoL during and in-between attacks. High AE-QoL scores at initial assessment and in placebo arms demonstrate the poor HRQoL among patients with HAE.
CONCLUSIONS: Only one study was identified that captured HRQoL during and between patients’ HAE attacks. There remains difficulty in accurately quantifying the impact of each attack on HRQoL, likely due to the transient and unpredictable nature of attacks. This highlights the need for further research to improve understanding of how to measure patient HRQoL during attacks.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR144
Topic
Economic Evaluation, Patient-Centered Research, Study Approaches
Topic Subcategory
Health State Utilities, Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases