{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,25]],"date-time":"2026-09-25T15:11:06Z","timestamp":1790349066510,"version":"4.1.0"},"reference-count":29,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2024,4,5]],"date-time":"2024-04-05T00:00:00Z","timestamp":1712275200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2024,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Despite the rapid uptake of emicizumab in the paediatric haemophilia A (HA) population, real\u2010world data on the safety and efficacy is limited.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To report on bleeding and safety in paediatric patients receiving emicizumab prophylaxis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Data were extracted from the multicentre prospective observational PedNet Registry (NCT02979119). Children with haemophilia A, and \u226550 FVIII exposures or inhibitors present receiving emicizumab maintenance therapy were analysed. Data were summarized as medians with interquartile range (IQR, P25\u2013P75). Mean (95% confidence interval (CI)), annualized (joint) bleeding rate (A(J)BR) during emicizumab and \u22642 years before emicizumab prophylaxis were modelled and compared using negative binomial regression.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Total of 177 patients started emicizumab at median 8.6 years (IQR 4.8\u201313.1), most had no FVIII inhibitors (64%). Follow up before emicizumab was median: 1.68 years (IQR: 1.24\u20131.90) and during emicizumab: 1.32 years (IQR:\u00a0.94\u20132.11).<\/jats:p><jats:p>In patients without inhibitors, mean ABR reduced after starting emicizumab from 2.41 (CI 1.98\u20132.95) to 1.11 (CI\u00a0.90\u20131.36, <jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.001), while AJBR reduced from.74 (CI\u00a0.56\u2013.98) to.31 (CI\u00a0.21\u2013.46, <jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.001). Concordantly, in patients with inhibitors, mean ABR reduced from 5.08 (CI 4.08\u20136.38) to\u00a0.75 (CI\u00a0.56\u20131.01, <jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.001), while AJBR reduced from 1.90 (CI 1.42\u20132.58) to\u00a0.34 (CI\u00a0.21\u2013.56, <jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.001). Five emicizumab\u2010related adverse events were reported (3% of the cohort), including one patient with antidrug antibodies.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>This study showed improved bleeding control compared to previous treatment and a favourable safety profile during emicizumab therapy in paediatric haemophilia A patients.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.15015","type":"journal-article","created":{"date-parts":[[2024,4,5]],"date-time":"2024-04-05T16:02:55Z","timestamp":1712332975000},"page":"685-692","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":18,"title":["Bleeding control improves after switching to emicizumab: Real\u2010world experience of 177 children in the PedNet registry"],"prefix":"10.1111","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9082-1416","authenticated-orcid":false,"given":"Konrad","family":"van der Zwet","sequence":"first","affiliation":[{"name":"Center for Benign Haematology Thrombosis and Haemostasis Van Creveldkliniek University Medical Center Utrecht Utrecht University  Utrecht The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3521-1863","authenticated-orcid":false,"given":"Marloes","family":"de Kovel","sequence":"additional","affiliation":[{"name":"PedNet Haemophilia Research Foundation  Baarn The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jayashree","family":"Motwani","sequence":"additional","affiliation":[{"name":"Department of Paediatric Haematology Birmingham Children's Hospital  Birmingham UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chris","family":"van Geet","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Leuven  Leuven Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Beatrice","family":"Nolan","sequence":"additional","affiliation":[{"name":"Children's Health Ireland at Crumlin  Dublin Ireland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heidi","family":"Glosli","sequence":"additional","affiliation":[{"name":"Centre for Rare Disorders Oslo University Hospital  Oslo Norway"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmen","family":"Escuriola Ettingshausen","sequence":"additional","affiliation":[{"name":"H\u00e4mophilie\u2010Zentrum Rhein Main  M\u00f6rfelden\u2010Walldorf Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christoph","family":"K\u00f6nigs","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Adolescents Medicine Clinical and Molecular Haemostasis University Hospital Frankfurt  Frankfurt Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1279-2557","authenticated-orcid":false,"given":"Gili","family":"Kenet","sequence":"additional","affiliation":[{"name":"National Hemophilia Center Sheba Medical Center  Tel Hashomer Israel"},{"name":"The Amalia Biron Thrombosis Research Institute Tel Aviv University  Tel Aviv Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7126-6613","authenticated-orcid":false,"given":"Kathelijn","family":"Fischer","sequence":"additional","affiliation":[{"name":"Center for Benign Haematology Thrombosis and Haemostasis Van Creveldkliniek University Medical Center Utrecht Utrecht University  Utrecht The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"the PedNet Investigators","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,4,5]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41572\u2010021\u201000278\u2010x"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14412"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1703068"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1803550"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352\u20103026(19)30054\u20107"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019001869"},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13848"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.16964"},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.1080\/08880018.2021.2004269"},{"key":"e_1_2_13_11_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2023021832"},{"key":"e_1_2_13_12_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12571"},{"key":"e_1_2_13_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13877"},{"key":"e_1_2_13_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14318"},{"key":"e_1_2_13_15_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.15342"},{"key":"e_1_2_13_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13762"},{"key":"e_1_2_13_17_1","doi-asserted-by":"publisher","DOI":"10.1080\/17474086.2021.1875817"},{"key":"e_1_2_13_18_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12448"},{"key":"e_1_2_13_19_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14847"},{"key":"e_1_2_13_20_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12613"},{"key":"e_1_2_13_21_1","doi-asserted-by":"publisher","DOI":"10.1160\/TH17\u201001\u20100059"},{"key":"e_1_2_13_22_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102516.2010.02383.x"},{"key":"e_1_2_13_23_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14432"},{"key":"e_1_2_13_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14826"},{"key":"e_1_2_13_25_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.rpth.2023.102180"},{"key":"e_1_2_13_26_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.rpth.2023.100074"},{"key":"e_1_2_13_27_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020009217"},{"key":"e_1_2_13_28_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12837"},{"key":"e_1_2_13_29_1","doi-asserted-by":"publisher","DOI":"10.1080\/03007995.2022.2105072"},{"key":"e_1_2_13_30_1","doi-asserted-by":"publisher","DOI":"10.1080\/17474086.2022.2112171"}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.15015","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,4,24]],"date-time":"2025-04-24T11:09:29Z","timestamp":1745492969000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.15015"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,4,5]]},"references-count":29,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2024,5]]}},"alternative-id":["10.1111\/hae.15015"],"URL":"https:\/\/doi.org\/10.1111\/hae.15015","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"value":"1351-8216","type":"print"},{"value":"1365-2516","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,4,5]]},"assertion":[{"value":"2024-01-09","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-03-31","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-04-05","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}