{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,2,11]],"date-time":"2025-02-11T05:24:10Z","timestamp":1739251450460,"version":"3.37.0"},"reference-count":26,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2024,12,12]],"date-time":"2024-12-12T00:00:00Z","timestamp":1733961600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004326","name":"Bayer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004326","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["European J of Haematology"],"published-print":{"date-parts":[[2025,3]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Objectives<\/jats:title><jats:p>To report the long\u2010term safety and efficacy of BAY 81\u20108973 in the LEOPOLD Kids extension phase.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Patients received BAY 81\u20108973 (25\u201350\u2009IU\/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (<jats:italic>n<\/jats:italic>\u2009=\u200982). Efficacy endpoints were assessed in patients without high\u2010titre inhibitors\/immune tolerance induction (<jats:italic>n<\/jats:italic>\u2009=\u200967).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Children (<jats:italic>n<\/jats:italic>\u2009=\u200982) received BAY 81\u20108973 for a median of 3.1\u2009years per patient and a median of 405 exposure days per patient. Long\u2010term BAY 81\u20108973 treatment was well tolerated, with no cases of de novo inhibitor development in the extension phase. Annualised bleeding rates (ABRs) within 48\u2009h of prophylaxis were low for all bleeds (median [IQR], 0.7 [0\u20131.9]; mean, 1.4 [SD, 2.1]) and for joint bleeds (median [IQR], 0 [0\u20130.7]; mean, 0.5 [SD, 1.1]) (<jats:italic>n<\/jats:italic>\u2009=\u200967). Twenty\u2010one of 67 patients (31.3%) had zero bleeds within 48\u2009h of prophylaxis; the treatment response was \u2018good\u2019\/\u2018excellent\u2019 in 87.9% of bleeds, and most bleeds resolved with \u2264\u20092 BAY 81\u20108973 infusions (83.5%).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Long\u2010term BAY 81\u20108973 treatment is well tolerated and maintains low ABRs for all bleeds and joint bleeds in children with severe haemophilia A.<\/jats:p><jats:p>\n<jats:bold>Trial Registration:<\/jats:bold> <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/clinicaltrials.gov\">ClinicalTrials.gov<\/jats:ext-link> identifier: NCT01311648<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.14362","type":"journal-article","created":{"date-parts":[[2024,12,13]],"date-time":"2024-12-13T03:05:07Z","timestamp":1734059107000},"page":"556-565","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["<scp>BAY<\/scp> 81\u20108973 Demonstrates Long\u2010Term Safety and Efficacy in Children With Severe Haemophilia A: Results From the <scp>LEOPOLD<\/scp> Kids Extension Study"],"prefix":"10.1111","volume":"114","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3999-8747","authenticated-orcid":false,"given":"Rolf","family":"Ljung","sequence":"first","affiliation":[{"name":"Department of Clinical Sciences Lund\u2010Pediatrics Lund University  Lund Sweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anthony K. 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Saulyte","family":"Trakymiene","sequence":"additional","affiliation":[{"name":"Clinic of Children's Diseases, Faculty of Medicine, Vilnius University Vilnius University Hospital Santaros Klinikos  Vilnius Lithuania"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Heidi","family":"Glosli","sequence":"additional","affiliation":[{"name":"Center for Rare Disorders and Department of Pediatric Research Oslo University Hospital  Oslo Norway"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gili","family":"Kenet","sequence":"additional","affiliation":[{"name":"Sheba Medical Center, The Israeli National Hemophilia Center, The Amalia Biron Thrombosis Research Institute and The Sackler Medical School Tel Aviv University  Tel\u2010Hashomer 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