{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T18:54:05Z","timestamp":1783018445745,"version":"3.54.6"},"reference-count":28,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2026,4,26]],"date-time":"2026-04-26T00:00:00Z","timestamp":1777161600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,4,26]],"date-time":"2026-04-26T00:00:00Z","timestamp":1777161600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100014935","name":"BioCryst","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014935","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Clin Experimental Allergy"],"published-print":{"date-parts":[[2026,7]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>In the phase 3 APeX\u20102 and APeX\u2010J trials, improvements in health\u2010related quality of life (HRQoL) were reported for berotralstat, the only oral, once\u2010daily, long\u2010term prophylaxis for hereditary angioedema (HAE). We conducted a post hoc trial analysis to evaluate the impact of berotralstat on HRQoL versus placebo through 24\u2009weeks, and the long\u2010term effects of treatment up to 96\u2009weeks, using pooled APeX\u20102 and APeX\u2010J patient data.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      Patients receiving berotralstat 150\u2009mg or placebo using the Angioedema QoL (AE\u2010QoL) questionnaire; total and domain scores were analysed. Changes from baseline through Week 24 were compared using mixed models for repeated measures (MMRM) to estimate least squares mean differences (LSMDs) with 95% confidence intervals (CI) and nominal\n                      <jats:italic>p<\/jats:italic>\n                      \u2010values. After Week 24, in the absence of a placebo comparator, long\u2010term changes from baseline were evaluated using within\u2010subject effect sizes (Cohen's\n                      <jats:italic>d<\/jats:italic>\n                      ) through Week 96 in patients receiving berotralstat. Clinical meaningful improvements in AE\u2010QoL total and domain scores were assessed by minimal clinically important difference (6\u2010point reduction in AE\u2010QoL total score) and distributional criterion methods, respectively. The manufacturer of berotralstat (BioCryst Pharmaceuticals) funded this study.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Overall, 47 and 45 patients received berotralstat and placebo, respectively. At Week 24, berotralstat showed significantly greater improvement in AE\u2010QoL total (LSMD: \u22127.4; 95% CI: \u221214.3, \u22120.6;\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.05), functioning domain (LSMD: \u221210.9; 95% CI: \u221219.6, \u22122.3;\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.013) and fears\/shame domain (LSMD: \u22128.7; 95% CI: \u221217.2, \u22120.2;\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.046) scores compared with placebo. Among patients receiving berotralstat, consistent sustained improvements were observed across all AE\u2010QoL domains up to Week 96. Analyses of meaningful improvement in total score and domains showed an increase over time in the proportion of patients achieving meaningful change.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Berotralstat significantly improved HRQoL versus placebo through 24\u2009weeks and showed long\u2010term beneficial effects through 96\u2009weeks.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/cea.70274","type":"journal-article","created":{"date-parts":[[2026,4,27]],"date-time":"2026-04-27T04:34:45Z","timestamp":1777264485000},"page":"730-738","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Berotralstat and Health\u2010Related Quality of Life in Hereditary Angioedema: Pooled Analysis of the\n                    <scp>APeX<\/scp>\n                    \u20102 and\n                    <scp>APeX<\/scp>\n                    \u2010J Trials"],"prefix":"10.1111","volume":"56","author":[{"given":"Teresa","family":"Caballero","sequence":"first","affiliation":[{"name":"Allergy Department Hospital Universitario La Paz, IdiPAZ, CIBERER U754  Madrid Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sorena","family":"Kiani\u2010Alikhan","sequence":"additional","affiliation":[{"name":"Department of Immunology Royal Free London NHS Foundation Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Douglas T.","family":"Johnston","sequence":"additional","affiliation":[{"name":"BioCryst Pharmaceuticals, Inc.  Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3490-648X","authenticated-orcid":false,"given":"Aolin","family":"Wang","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc.  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lorena","family":"Lopez\u2010Gonzalez","sequence":"additional","affiliation":[{"name":"BioCryst Pharmaceuticals, Inc.  Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9014-8479","authenticated-orcid":false,"given":"Zheng\u2010Yi","family":"Zhou","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc.  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fan","family":"Yang","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc.  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9640-7299","authenticated-orcid":false,"given":"Hannah H.","family":"Kim","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc.  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra","family":"Nestler\u2010Parr","sequence":"additional","affiliation":[{"name":"BioCryst Pharmaceuticals, Inc.  Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dianne","family":"Tomita","sequence":"additional","affiliation":[{"name":"BioCryst Pharmaceuticals, Inc.  Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick","family":"Gillard","sequence":"additional","affiliation":[{"name":"BioCryst Pharmaceuticals, Inc.  Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tamar","family":"Kinaciyan","sequence":"additional","affiliation":[{"name":"Department of Dermatology Medical University of Vienna  Vienna Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,4,26]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.12968\/hmed.2019.80.7.391"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm10092023"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-024-03265-z"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-025-03539-0"},{"key":"e_1_2_12_6_1","unstructured":"\u201cBioCryst Announces FDA Approval of ORLADEYO (Berotralstat) as the First Oral Once\u2010Daily Therapy to Prevent Attacks of Hereditary Angioedema (HAE) \u201d (2020)."},{"key":"e_1_2_12_7_1","unstructured":"Orladeyo \u201cHighlights of Prescribing Information 2020 \u201dhttps:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2020\/214094s000lbl.pdf."},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2020.10.015"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1002\/clt2.12035"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.waojou.2024.100882"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaip.2021.03.057"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/all.14670"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/all.12007"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/all.12900"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.3389\/fpsyg.2013.00863"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1097\/01.MLR.0000062554.74615.4C"},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.anai.2020.02.018"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.2500\/aap.2014.35.3738"},{"key":"e_1_2_12_19_1","doi-asserted-by":"publisher","DOI":"10.2500\/aap.2013.34.3685"},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.2500\/aap.2010.31.3394"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.anai.2013.05.028"},{"key":"e_1_2_12_22_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-9-99"},{"key":"e_1_2_12_23_1","doi-asserted-by":"publisher","DOI":"10.1111\/all.15214"},{"key":"e_1_2_12_24_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2023.11.292"},{"key":"e_1_2_12_25_1","unstructured":"D. F. C. K.SoteresandS.Czado \u201cAssessing the Burden of Injection With Parenteral Hereditary Angioedema Therapies \u201dPresented at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Boston MA May 7\u201310 2023.2023."},{"key":"e_1_2_12_26_1","doi-asserted-by":"publisher","DOI":"10.2500\/aap.2021.42.210017"},{"key":"e_1_2_12_27_1","doi-asserted-by":"publisher","DOI":"10.1186\/s41687-025-00876-3"},{"key":"e_1_2_12_28_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaip.2025.06.018"},{"key":"e_1_2_12_29_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2024.12.895"}],"container-title":["Clinical &amp; Experimental Allergy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/cea.70274","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/cea.70274","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/cea.70274","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T17:45:49Z","timestamp":1783014349000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/cea.70274"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,4,26]]},"references-count":28,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2026,7]]}},"alternative-id":["10.1111\/cea.70274"],"URL":"https:\/\/doi.org\/10.1111\/cea.70274","archive":["Portico"],"relation":{},"ISSN":["0954-7894","1365-2222"],"issn-type":[{"value":"0954-7894","type":"print"},{"value":"1365-2222","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,4,26]]},"assertion":[{"value":"2025-10-22","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-02-12","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-04-26","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}