{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,8]],"date-time":"2026-10-08T10:55:20Z","timestamp":1791456920695,"version":"4.3.4"},"reference-count":24,"publisher":"Oceanside Publications Inc.","issue":"4","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["allergy asthma proc"],"published-print":{"date-parts":[[2024,7,1]]},"abstract":"<jats:p><jats:bold>Background:<\/jats:bold> Hereditary angioedema (HAE) is a rare genetic condition characterized by painful and often debilitating swelling attacks. Little is known about the differences in outcomes between patients with HAE types I or II (type I: HAE caused by C1 esterase inhibitor deficiency;\n type II: HAE caused by C1 esterase inhibitor dysfunction), with decreased or dysfunctional C1 esterase inhibitor (C1-INH), and those with normal C1-INH (nC1-INH-HAE).  <jats:bold>Objective:<\/jats:bold> To compare physician- and patient-reported real-world outcomes in patients with HAE types I\/II\n versus patients with nC1-INH-HAE.  <jats:bold>Methods:<\/jats:bold> Data were drawn from the Adelphi HAE Disease Specific Programme<jats:sup>TM<\/jats:sup> a real-world, cross-sectional survey of HAE-treating physicians and their patients in the United States conducted between July and November 2021. Physicians\n reported patient disease activity and severity, and recent attack history. Patient-reported outcomes were collected. Bivariate tests used were either the Student\u2019s <jats:italic>t<\/jats:italic>-test, the Fisher exact test, or Mann-Whitney U test.  <jats:bold>Results:<\/jats:bold> Physicians (N = 67) provided\n data on 368 patients (92.4% HAE types I\/II and 7.6% nC1-INH-HAE). Physicians reported that a higher proportion of patients with nC1-INH-HAE had moderate or high disease activity and moderate or severe disease severity both at diagnosis and at data collection versus those with HAE types I\/II.\n Patients with nC1-INH-HAE versus patients with HAE types I\/II experienced increased attack severity (34.6% versus 4.4%) and hospitalization rate during the most recent attack (39.3% versus 6.6%), and reported lower health status and quality of life, <jats:italic>via<\/jats:italic> the European Quality of Life\n 5 Dimension 5 Level (US tariff) and Angioedema Quality of Life, respectively. On average, 25% of the patients with nC1-INH-HAE reported absenteeism and work or activity impairment due to HAE compared with 2.7% of patients with HAE types I\/II. Both patient groups reported improvements in disease\n activity and severity from diagnosis to the time of data collection.  <jats:bold>Conclusion:<\/jats:bold> These real-world findings suggest that patients with nC1-INH-HAE have increased disease activity and severity, and experience greater impairment to their quality of life, work, and daily functioning\n than patients with HAE types I\/II. Powered statistical analyses are required to confirm these findings.<\/jats:p>","DOI":"10.2500\/aap.2024.45.240021","type":"journal-article","created":{"date-parts":[[2024,7,10]],"date-time":"2024-07-10T04:33:48Z","timestamp":1720586028000},"page":"247-254","source":"Crossref","is-referenced-by-count":15,"title":["Physician- and patient-reported outcomes by hereditary angioedema type: Data from a real-world study"],"prefix":"10.2500","volume":"45","author":[{"given":"John","family":"Anderson","sequence":"first","affiliation":[{"name":"From Clinical Research, AllerVie Health, Birmingham, Alabama"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Soteres","sequence":"additional","affiliation":[{"name":"Allergy\/Immunology, Asthma and Allergy Associates, Colorado Springs, Colorado"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Mellor","sequence":"additional","affiliation":[{"name":"Rare Disease Franchise, Adelphi Real World, Bollington, United Kingdom, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hannah","family":"Connolly","sequence":"additional","affiliation":[{"name":"Rare Disease Franchise, Adelphi Real World, Bollington, United Kingdom, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kieran","family":"Wynne-Cattanach","sequence":"additional","affiliation":[{"name":"Rare Disease Franchise, Adelphi Real World, Bollington, United Kingdom, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lucy","family":"Earl","sequence":"additional","affiliation":[{"name":"Rare Disease Franchise, Adelphi Real World, Bollington, United Kingdom, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bob G.","family":"Schultz","sequence":"additional","affiliation":[{"name":"Takeda Pharmaceuticals USA, Inc., Lexington, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Salome","family":"Juethner","sequence":"additional","affiliation":[{"name":"Takeda Pharmaceuticals USA, Inc., Lexington, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1355","reference":[{"key":"B1","doi-asserted-by":"crossref","first-page":"1961","DOI":"10.1111\/all.15214","article-title":"The international WAO\/EAACI guideline for the management of hereditary angioedema\u2014the 2021 revision and update","volume":"77","author":"Maurer","year":"2022","journal-title":"Allergy"},{"key":"B2","first-page":"S292","article-title":"Severity of hereditary angioedema, prevalence, and diagnostic considerations","volume":"24","author":"Bernstein","year":"2018","journal-title":"Am J Manag Care"},{"key":"B3","doi-asserted-by":"crossref","first-page":"297","DOI":"10.3949\/ccjm.80a.12073","article-title":"Recognizing and managing hereditary angioedema","volume":"80","author":"Tse","year":"2013","journal-title":"Cleve Clin J Med"},{"key":"B4","doi-asserted-by":"crossref","first-page":"391","DOI":"10.12968\/hmed.2019.80.7.391","article-title":"Hereditary angioedema: an update on causes, manifestations and treatment","volume":"80","author":"Longhurst","year":"2019","journal-title":"Br J Hosp Med (Lond)"},{"key":"B5","doi-asserted-by":"crossref","first-page":"441","DOI":"10.2500\/aap.2019.40.4267","article-title":"Hereditary and acquired angioedema","volume":"40","author":"Patel","year":"2019","journal-title":"Allergy Asthma Proc"},{"key":"B6","doi-asserted-by":"crossref","first-page":"2229","DOI":"10.1016\/j.jaip.2021.03.008","article-title":"The expanding spectrum of mutations in hereditary angioedema","volume":"9","author":"Veronez","year":"2021","journal-title":"J Allergy Clin Immunol Pract"},{"key":"B7","doi-asserted-by":"crossref","first-page":"600","DOI":"10.1016\/j.anai.2020.02.018","article-title":"Patient-reported burden of hereditary angioedema: findings from a patient survey in the United States","volume":"124","author":"Banerji","year":"2020","journal-title":"Ann Allergy Asthma Immunol"},{"key":"B8","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1186\/1750-1172-9-99","article-title":"Socioeconomic burden of hereditary angioedema: results from the hereditary angioedema burden of illness study in Europe","volume":"9","author":"Ayg\u00f6ren-P\u00fcrs\u00fcn","year":"2014","journal-title":"Orphanet J Rare Dis"},{"key":"B9","doi-asserted-by":"crossref","first-page":"47","DOI":"10.2500\/aap.2013.34.3685","article-title":"The humanistic burden of hereditary angioedema: results from the Burden of Illness Study in Europe","volume":"35","author":"Caballero","year":"2014","journal-title":"Allergy Asthma Proc"},{"key":"B10","doi-asserted-by":"crossref","first-page":"371","DOI":"10.1016\/j.anai.2013.05.028","article-title":"Depression and anxiety in patients with hereditary angioedema","volume":"112","author":"Fouche","year":"2014","journal-title":"Ann Allergy Asthma Immunol"},{"key":"B11","doi-asserted-by":"crossref","first-page":"1575","DOI":"10.1111\/all.13384","article-title":"The International WAO\/EAACI Guideline for the Management of Hereditary Angioedema\u2014the 2017 revision and update","volume":"73","author":"Maurer","year":"2018","journal-title":"Allergy"},{"key":"B12","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1016\/j.jaip.2020.08.046","article-title":"US HAEA Medical Advisory Board 2020 Guidelines for the Management of Hereditary Angioedema","volume":"9","author":"Busse","year":"2021","journal-title":"J Allergy Clin Immunol Pract"},{"key":"B13","doi-asserted-by":"crossref","first-page":"72","DOI":"10.1186\/s13223-019-0376-8","article-title":"The International\/Canadian Hereditary Angioedema Guideline","volume":"15","author":"Betschel","year":"2019","journal-title":"Allergy Asthma Clin Immunol"},{"key":"B14","doi-asserted-by":"crossref","first-page":"814","DOI":"10.1007\/s12325-022-02401-0","article-title":"Hereditary angioedema: diagnosis, clinical implications, and pathophysiology","volume":"40","author":"Sinnathamby","year":"2023","journal-title":"Adv Ther"},{"key":"B15","doi-asserted-by":"crossref","first-page":"1136","DOI":"10.1056\/NEJMra1808012","article-title":"Hereditary angioedema","volume":"382","author":"Busse","year":"2020","journal-title":"N Engl J Med"},{"key":"B16","doi-asserted-by":"crossref","first-page":"716","DOI":"10.1016\/j.jaip.2021.11.011","article-title":"Specific targeting of plasma kallikrein for treatment of hereditary angioedema: a revolutionary decade","volume":"10","author":"Busse","year":"2022","journal-title":"J Allergy Clin Immunol Pract"},{"key":"B17","doi-asserted-by":"crossref","first-page":"320","DOI":"10.1111\/all.13076","article-title":"Treatment for hereditary angioedema with normal C1-INH and specific mutations in the F12 gene (HAE-FXII)","volume":"72","author":"Bork","year":"2017","journal-title":"Allergy"},{"key":"B18","doi-asserted-by":"crossref","first-page":"129","DOI":"10.1016\/j.jaci.2009.03.038","article-title":"Hereditary angioedema caused by missense mutations in the factor XII gene: clinical features, trigger factors, and therapy","volume":"124","author":"Bork","year":"2009","journal-title":"J Allergy Clin Immunol"},{"key":"B19","doi-asserted-by":"crossref","first-page":"475","DOI":"10.1111\/cea.12055","article-title":"Benefits of progestin contraception in non-allergic angioedema","volume":"43","author":"Saule","year":"2013","journal-title":"Clin Exp Allergy"},{"key":"B20","year":"2022"},{"key":"B21","doi-asserted-by":"crossref","first-page":"1727","DOI":"10.1007\/s11136-011-9903-x","article-title":"Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L)","volume":"20","author":"Herdman","year":"2011","journal-title":"Qual Life Res"},{"key":"B22","doi-asserted-by":"crossref","first-page":"1289","DOI":"10.1111\/all.12007","article-title":"Development and construct validation of the angioedema quality of life questionnaire","volume":"67","author":"Weller","year":"2012","journal-title":"Allergy"},{"key":"B23","doi-asserted-by":"crossref","first-page":"1203","DOI":"10.1111\/all.12900","article-title":"The Angioedema Quality of Life Questionnaire (AE-QoL) - assessment of sensitivity to change and minimal clinically important difference","volume":"71","author":"Weller","year":"2016","journal-title":"Allergy"},{"key":"B24","doi-asserted-by":"crossref","first-page":"353","DOI":"10.2165\/00019053-199304050-00006","article-title":"The validity and reproducibility of a work productivity and activity impairment instrument","volume":"4","author":"Reilly","year":"1993","journal-title":"Pharmacoeconomics"}],"container-title":["Allergy and Asthma Proceedings"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.ingentaconnect.com\/content\/ocean\/aap\/2024\/00000045\/00000004\/art00011","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,7,10]],"date-time":"2024-07-10T04:35:31Z","timestamp":1720586131000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.ingentaconnect.com\/content\/10.2500\/aap.2024.45.240021"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,7,1]]},"references-count":24,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2024,7,1]]}},"alternative-id":["1088-5412(20240701)45:4L.247;1-"],"URL":"https:\/\/doi.org\/10.2500\/aap.2024.45.240021","relation":{},"ISSN":["1088-5412"],"issn-type":[{"value":"1088-5412","type":"print"}],"subject":[],"published":{"date-parts":[[2024,7,1]]}}}