{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T17:50:15Z","timestamp":1792173015892,"version":"4.4.0"},"reference-count":42,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T00:00:00Z","timestamp":1791504000000},"content-version":"vor","delay-in-days":8,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100008322","name":"CSL Behring","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008322","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["ascpt.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Clinical Translational Sci"],"published-print":{"date-parts":[[2026,10]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>\n                    Hereditary angioedema (HAE) attacks are recurrent, debilitating, and potentially life\u2010threatening. Across the clinical program, garadacimab long\u2010term prophylaxis demonstrated early onset and durable, sustained protection against HAE attacks and clinically meaningful improvements in health\u2010related quality of life (HRQoL). HAE treatment goals include total disease control (attack\u2010free) and normalization of life. Predicting treatment effect may aid shared decision\u2010making and choice of long\u2010term prophylaxis. Here, we report an attack\u2010free probability model predicting garadacimab treatment effect beyond available Phase 3 clinical data and explore the relationship between HRQoL and attack\u2010free duration. A generalized mixed\u2010effect Poisson model was developed and validated with data from the garadacimab pivotal Phase 3 (VANGUARD) and Phase 3 open\u2010label extension studies. Efficacy variables, including attack\u2010free probabilities, were predicted for simulated populations of garadacimab\u2010treated patients and assessed for goodness of fit. Analysis of covariance was used to assess the relationship between Angioedema Quality of Life questionnaire scores and attack\u2010free duration. Model\u2010predicted efficacy variables adequately reflected observed clinical data with high goodness of fit (Nakagawa\u2013Schielzeth conditional\n                    <jats:italic>R<\/jats:italic>\n                    <jats:sup>2<\/jats:sup>\n                    : 74.3%). Following a 1\u2010month attack\u2010free period after garadacimab initiation, patients had a 60% cumulative probability of remaining attack\u2010free up to Month\u200925 and 49% up to 5\u2009years. HRQoL improved with increasing duration of attack\u2010free periods, reaching minimal to no impairment after 6\u2009months. This attack\u2010free probability model suggests long\u2010term effects of garadacimab over a 5\u2010year treatment period. Patients treated with garadacimab have the potential to live attack\u2010free for long periods of time, with substantial HRQoL improvements.\n                  <\/jats:p>","DOI":"10.1111\/cts.70737","type":"journal-article","created":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T12:29:01Z","timestamp":1791548941000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Garadacimab in Hereditary Angioedema: Modeling Suggests Sustained Improvements in Attack\u2010Free Probability and Quality of Life Over 5\u2009Years"],"prefix":"10.1111","volume":"19","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9155-9162","authenticated-orcid":false,"given":"Philip H.","family":"Li","sequence":"first","affiliation":[{"name":"Division of Rheumatology and Clinical Immunology, Department of Medicine, Queen Mary Hospital University of Hong Kong  Hong Kong China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2201-9442","authenticated-orcid":false,"given":"F. Ida","family":"Hsu","sequence":"additional","affiliation":[{"name":"Section of Rheumatology, Allergy &amp; Immunology, Department of Medicine Yale University School of Medicine  New Haven Connecticut USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7763-4982","authenticated-orcid":false,"given":"Emel","family":"Ayg\u00f6ren\u2010P\u00fcrs\u00fcn","sequence":"additional","affiliation":[{"name":"University Hospital Frankfurt, Department for Children and Adolescents Goethe University Frankfurt  Frankfurt Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7608-5416","authenticated-orcid":false,"given":"William R.","family":"Lumry","sequence":"additional","affiliation":[{"name":"AARA Research Center  Dallas Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-2619-7239","authenticated-orcid":false,"given":"Jason","family":"Raasch","sequence":"additional","affiliation":[{"name":"Midwest Immunology Clinic  Plymouth Minnesota USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1259-5422","authenticated-orcid":false,"given":"Ingo","family":"Pragst","sequence":"additional","affiliation":[{"name":"CSL Innovation GmbH  Marburg Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0006-5866-8160","authenticated-orcid":false,"given":"Yinglei","family":"Li","sequence":"additional","affiliation":[{"name":"CSL Behring  King of Prussia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2319-2909","authenticated-orcid":false,"given":"Chiara","family":"Nenci","sequence":"additional","affiliation":[{"name":"CSL Behring AG  Bern Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4373-1669","authenticated-orcid":false,"given":"Marco","family":"Campioni","sequence":"additional","affiliation":[{"name":"CSL Vifor Pharma Management Ltd.  Zurich Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9218-5468","authenticated-orcid":false,"given":"Markus","family":"Magerl","sequence":"additional","affiliation":[{"name":"Charit\u00e9 \u2010 University Medical Center Berlin, Corporate Member of Freie Universit\u00e4t Berlin and Humboldt\u2010Universit\u00e4t Zu Berlin Institute of Allergology  Berlin Germany"},{"name":"Immunology and Allergology Fraunhofer Institute for Translational Medicine and Pharmacology ITMP  Berlin Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,10,9]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1808012"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/all.15214"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.2500\/aap.2025.46.250017"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.2500\/aap.2025.46.250028"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010025\u201003539\u20100"},{"key":"e_1_2_10_7_1","unstructured":"ANDEMBRY FDA \u201cANDEMBRY (garadacimab\u2010gxii) Injection for Subcutaneous Use \u201d(2026) https:\/\/labeling.cslbehring.com\/PI\/US\/Andembry\/EN\/Andembry\u2010Prescribing\u2010Information.pdf."},{"key":"e_1_2_10_8_1","unstructured":"DAWNZERA FDA \u201cDAWNZERA (donidalorsen) Injection for Subcutaneous Use \u201d(2026) https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2025\/219407s000lbl.pdf."},{"key":"e_1_2_10_9_1","unstructured":"EKTERLYandFood and Drug Administration \u201cEKTERLY (sebetralstat) Tablets for Oral Use \u201d(2026) https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2025\/219301s000lbl.pdf."},{"key":"e_1_2_10_10_1","unstructured":"ANDEMBRY EMA \u201cANDEMBRY (garadacimab) EU Summary of Product Characteristics 2025 \u201d(2026) https:\/\/www.ema.europa.eu\/en\/documents\/product\u2010information\/andembry\u2010epar\u2010product\u2010information_en.pdf."},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010025\u201003739\u20108"},{"key":"e_1_2_10_12_1","unstructured":"ANDEMBRY Australian Therapeutic Goods Administration \u201cANDEMBRY (garadacimab) Solution for Subcutaneous Injection. 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