{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,14]],"date-time":"2026-05-14T05:10:54Z","timestamp":1778735454491,"version":"3.51.4"},"reference-count":33,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2026,5,14]],"date-time":"2026-05-14T00:00:00Z","timestamp":1778716800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Immunol."],"abstract":"<jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Angioedema due to acquired C1 inhibitor deficiency (AAE-C1INH) is a rare, serious condition that manifests with recurring and often painful swelling attacks. No approved treatments or validated patient-reported outcome (PRO) measures exist for AAE-C1INH. This study aimed to develop a conceptual model of AAE-C1INH and explored the relevance of adapting PRO measures validated for hereditary angioedema, another bradykinin-mediated disease.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This cross-sectional, qualitative study involved semi-structured interviews with 8 adults living with AAE-C1INH. Open-ended questions elicited participants\u2019 descriptions of AAE-C1INH manifestations and daily life impacts. Cognitive interviews assessed perceptions of clarity, comprehension, and meaningful levels of change in PRO measures: Patient Global Impression of Change (PGI-C) and Severity (PGI-S) and Patient Global Assessment of Status (PGA-S) and Change (PGA-C).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Participants reported traumatic medical emergencies, misdiagnoses, and evaluations by a variety of healthcare providers. Daily life impact was common (social\/family and treatment-related; n=7). Attack areas most frequently reported were abdomen (n=7), face (n=6), and foot\/hand (n=5 each). Evaluating PGI-C, 8\/8 participants correctly interpreted the scale to assess symptoms at a given time post-treatment vs. at time of treatment for a theoretical AAE-C1INH attack. Four of 8 participants considered \u201ca little better\u201d \u22652 hours post-treatment and 6\/6 considered \u201cbetter\u201d \u22654 hours as meaningful. Most also considered PGI-S and PGA easy to interpret and relevant to their symptoms.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Interviews highlighted the impact of AAE-C1INH on participants\u2019 lives and overall well-being. Participants\u2019 assessments of PRO items have informed the clinical outcome assessment strategy for the first phase 3 clinical trial exclusively in AAE-C1INH.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3389\/fimmu.2026.1810851","type":"journal-article","created":{"date-parts":[[2026,5,14]],"date-time":"2026-05-14T04:29:47Z","timestamp":1778732987000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":0,"title":["Angioedema due to acquired C1 inhibitor deficiency: patient experience, conceptual disease model, and assessment of patient-reported outcome measures"],"prefix":"10.3389","volume":"17","author":[{"given":"Mats","family":"de Lange","sequence":"first","affiliation":[{"name":"Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam","place":["Amsterdam, Netherlands"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Danny 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