Background: Hereditary angioedema (HAE) attacks substantially impair health-related quality of life (HRQoL). Current World Allergy Organization and the European Academy of Allergy and Clinical Immunology guidelines goals include complete control and normalization of patients' lives. Garadacimab (anti-activated factor XII monoclonal antibody) reduced the mean attack rate after first administration in the pivotal phase III (VANGUARD; NCT04656418) and ongoing long-term phase III open-label extension (OLE) (NCT04739059) studies. Objective: To report exploratory HRQoL data from the interim analysis of the phase III OLE study (data cutoff February 13, 2023). Methods: Patients ages ≥12 years and with HAE received garadacimab 200 mg subcutaneously once monthly in the OLE study. The patient population comprised patients who were garadacimab naive (received placebo in the previous phase III study and newly enrolled patients) and patients who received garadacimab in previous phase II/III studies. The Angioedema Quality of Life (AE-QoL) questionnaire, Treatment Satisfaction Questionnaire for Medication version II (TSQM II), and Work Productivity and Activity Impairment: General Health (WPAI:GH) questionnaire were administered at baseline and every 3 months during the OLE study. AE-QoL and TSQM II scores were evaluated in comparison with minimal clinically important differences (MCID). Results: Overall, 90 patients who were garadacimab naive and 71 patients with previous garadacimab exposure received garadacimab in the phase III OLE study. The mean ± standard deviation AE-QoL total score improved by 34.2 ± 18.8 points in patients who were garadacimab naive and by 2.3 ± 13.1 points further to the reduction experienced in patients with previous garadacimab exposure. The AE-QoL MCID was met by 92.1% of patients who were garadacimab naive; 81.6% of patients with previous garadacimab exposure experienced stable AE-QoL scores or further improvements per MCID. TSQM II scores were improved from day 1 with garadacimab and sustained to month 12. Improvements in WPAI:GH scores were consistent with AE-QoL and TSQM II. Conclusion: Garadacimab elicited clinically meaningful long-term improvements in HRQoL, work productivity, and treatment satisfaction in patients with HAE, which brought them closer to complete disease control and normalization of life.Clinical trial NCT04739059, clinicaltrials.gov.

Allergy and Asthma Research Associates Research Center Dallas Texas

Allergy Immunology and Angioedema Center Barzilai University Hospital Ashkelon Israel

Angioedema Center of Reference and Excellence Institute of Allergology Charité Universitätsmedizin Berlin Freie Universität Berlin and Humboldt Universität Zu Berlin Berlin Germany

CSL Behring AG Bern Switzerland

CSL Behring King of Prussia Pennsylvania

CSL Innovation GmbH Marburg Germany

Department for Hemotology Oncology Hämophilie Zentrum Rhein Main GmbH Mörfelden Walldorf Germany

Department of Immunology Charles University and University Hospital Motol Prague Czech Republic

From the Allergy Department Hospital Universitari Vall d'Hebron Vall d'Hebron Research Institute Barcelona Spain

Immunology and Allergy Unit Dept of Medicine Campbelltown Hospital and Western Sydney University Sydney New South Wales Australia

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