Rationale and design of the Garadacimab REAl-world treatment outcomes of effec-tiveness, safety and quality-of-life in patients with hereditary angioedema (GREAT) study.
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All Authors
Zanichelli A.
Magerl M.
Bouillet L.
Betschel S.D.
Savic S.
Nenci C.
Katkade V.
Riedl M.A.
LTHT Author
Savic, Sinisa
LTHT Department
Pathology
Clinical Immunology & Allergy
Clinical Immunology & Allergy
Non Medic
Publication Date
2025
Item Type
Conference Abstract
Language
Subject
ANGIOEDEMA , QUALITY OF LIFE , BIOMEDICAL RESEARCH
Subject Headings
Abstract
Background: Garadacimab (anti-activated factor XII antibody) has demonstrated early and durable efficacy with a favourable long-term safety profile in the Phase 2, pivotal Phase 3 (VANGUARD) and ongoing Phase 3 open-label extension studies in patients with hereditary angioedema (HAE). In 2025, garadacimab was approved for use as once-monthly long-term prophylaxis (LTP) in patients with HAE in Europe, the UK, Australia and Japan. Real-world data provide insights from routine clinical practice and can be used to inform treatment decisions in HAE. The GREAT study will investigate long-term effectiveness, safety, health-related quality of life (HRQoL) and healthcare resource utilisation (HCRU) outcomes in patients with HAE receiving garadacimab LTP in the real-world setting. Material(s) and Method(s): This prospective, noninterventional, observational cohort study will enrol patients aged >= 12 years with physician- and laboratory-confirmed HAE newly initiating on-label garadacimab (200 mg subcutaneous once monthly) from ~ 30 centres in Europe, the UK, North America and the Asia.Pacific region (target enrolment: 200 patients). Participants will be observed on treatment for a target duration of 24 months with a 30-day follow-up period. The GREAT study will evaluate the characteristics of the enrolled population, HAE attack rate (on treatment with garadacimab vs pre-enrolment baseline), time to occurrence of first HAE attack, the proportion of patients achieving attack-free status and attack-free duration. It will assess safety, HRQoL, productivity, treatment patterns, HCRU and the economic impact of garadacimab LTP, treatment adherence and satisfaction, and patient preference. Retrospective data on HAE attacks and treatments, and on HCRU will be collected from 12 months before garadacimab initiation. Result(s): The study will begin to enrol patients in mid-2025 and is estimated to complete in 2029. Conclusion(s): The GREAT study will provide the first comprehensive real-world evidence on the use of garadacimab as LTP against HAE.
Journal
Allergy, Asthma and Clinical Immunology