{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,21]],"date-time":"2026-10-21T19:08:10Z","timestamp":1792609690720,"version":"4.5.0"},"reference-count":19,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2024,9,19]],"date-time":"2024-09-19T00:00:00Z","timestamp":1726704000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000050","name":"National Heart, Lung, and Blood Institute","doi-asserted-by":"publisher","award":["T32HL007149\u201044"],"award-info":[{"award-number":["T32HL007149\u201044"]}],"id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000050","name":"National Heart, Lung, and Blood Institute","doi-asserted-by":"publisher","award":["K99HL150595"],"award-info":[{"award-number":["K99HL150595"]}],"id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2024,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>The management of bleeding events (BEs) in haemophilia A (HA) and B (HB) patients with inhibitors necessitates the use of bypassing agents. The recombinant factor VIIa bypassing agent eptacog beta has demonstrated efficacy at treating BEs and managing perioperative bleeding in adults in phase three clinical studies.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To provide real\u2010world descriptions of eptacog beta use for BE treatment in patients on emicizumab or eptacog beta prophylaxis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This is a retrospective case series of 14 patients who received eptacog beta at seven haemophilia treatment centres, with HA (<jats:italic>n<\/jats:italic>\u00a0=\u00a011) or HB (<jats:italic>n<\/jats:italic>\u00a0=\u00a03) and inhibitors or anaphylaxis to factor replacement.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Twenty\u2010four spontaneous and traumatic BEs are described (muscle hematomas, joint hemarthroses, port site, and epistaxis) involving 11 subjects. Eptacog beta was effective for acute bleed treatment as both first\u2010line therapy and for treatment of BEs refractory to eptacog alfa in 23\/24 events. When eptacog beta was used for prophylaxis, 2\/3 patients reported a decreased frequency of breakthrough BEs compared with prophylactic eptacog alfa and one patient experienced a similar frequency of breakthrough BEs compared with prophylactic activated prothrombin complex concentrate. Eptacog beta provided effective bleed control for three subjects who underwent minor surgical procedures. Treatment with eptacog beta was estimated to be 46%\u201372% more cost\u2010effective than eptacog alfa. No safety concerns or adverse events were reported.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>In this case series, eptacog beta was safe, effective, and economical as first\u2010line therapy, treatment of refractory BEs, management of perioperative bleeding, or prophylaxis in haemophilia patients with inhibitors.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.15094","type":"journal-article","created":{"date-parts":[[2024,9,19]],"date-time":"2024-09-19T10:22:39Z","timestamp":1726741359000},"page":"1321-1331","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Real\u2010world effectiveness of eptacog beta in patients with haemophilia and inhibitors: A multi\u2010institutional case series"],"prefix":"10.1111","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3188-4465","authenticated-orcid":false,"given":"Kimberley","family":"Youkhana","sequence":"first","affiliation":[{"name":"University of North Carolina School of Medicine  Capel Hill North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0122-9320","authenticated-orcid":false,"given":"Glaivy","family":"Batsuli","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Emory University and Children's Healthcare of Atlanta  Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suchitra","family":"Acharya","sequence":"additional","affiliation":[{"name":"Cohen Children's Medical Center, Northwell Health, New Hyde Park  New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Osman","family":"Khan","sequence":"additional","affiliation":[{"name":"Oklahoma Center for Bleeding and Clotting Disorders at OU Health  Oklahoma City Oklahoma USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6347-4452","authenticated-orcid":false,"given":"Duc Q.","family":"Tran","sequence":"additional","affiliation":[{"name":"Department of Hematology and Medical Oncology, and the Hemophilia of Georgia Center for Bleeding &amp; Clotting Disorders Emory University  Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea","family":"Dvorak","sequence":"additional","affiliation":[{"name":"State University of New York Upstate Medical Center  Syracuse New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Recht","sequence":"additional","affiliation":[{"name":"Yale University School of Medicine  New Haven Connecticut USA"},{"name":"National Hemophilia Foundation  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6013-1254","authenticated-orcid":false,"given":"Guy","family":"Young","sequence":"additional","affiliation":[{"name":"Children's Hospital Los Angeles, University of Southern California  Los Angeles California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9509-9415","authenticated-orcid":false,"suffix":"Jr.","given":"Robert","family":"Sidonio","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Emory University and Children's Healthcare of Atlanta  Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7278-7357","authenticated-orcid":false,"given":"Yasmina","family":"Abajas","sequence":"additional","affiliation":[{"name":"University of North Carolina School of Medicine  Capel Hill North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,9,19]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/ejh.13193"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.239160"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019000658"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1703068"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1803550"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-0038-1660817"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12634"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2009.02012.x"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2006-04-017988"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2004.00912.x"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.18553\/jmcp.2022.21197"},{"key":"e_1_2_10_13_1","volume-title":"Package Insert","author":"SEVENFACT\u00ae"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13110"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13301"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14419"},{"key":"e_1_2_10_17_1","unstructured":"IMB.https:\/\/www.ibm.com\/us\u2010en\/marketplace\/micromedex\u2010red\u2010book"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.2147\/JBM.S61859"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0268-960X(08)70001-8"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14944"}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.15094","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,12,20]],"date-time":"2024-12-20T04:59:50Z","timestamp":1734670790000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.15094"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,9,19]]},"references-count":19,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2024,11]]}},"alternative-id":["10.1111\/hae.15094"],"URL":"https:\/\/doi.org\/10.1111\/hae.15094","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"value":"1351-8216","type":"print"},{"value":"1365-2516","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,9,19]]},"assertion":[{"value":"2024-05-16","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-08-25","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-09-19","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}