{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,5]],"date-time":"2026-08-05T06:52:40Z","timestamp":1785912760602,"version":"3.56.0"},"reference-count":18,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2023,8,30]],"date-time":"2023-08-30T00:00:00Z","timestamp":1693353600000},"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":[[2023,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Haemophilia A care has changed with the introduction of emicizumab. Experience on the youngest children is still scarce and clinical practice varies between haemophilia treatment centres.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>We aimed to assess the current clinical practice on emicizumab prophylaxis within PedNet, a collaborative research platform for paediatricians treating children with haemophilia.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>An electronic survey was sent to all PedNet members (<jats:italic>n<\/jats:italic>\u00a0=\u00a032) between October 2022 and February 2023. The survey included questions on the availability of emicizumab, on the practice of initiating prophylaxis in previously untreated or minimally treated patients (PUPs or MTPs) and emicizumab use in patients with or without inhibitors.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>All but four centres (28\/32; 88%) responded. Emicizumab was available in clinical practice in 25\/28 centres (89%), and in 3\/28 for selected patients only (e.g. with inhibitors). Emicizumab was the preferred choice for prophylaxis in PUPs or MTPs in 20\/25 centres; most (85%) started emicizumab prophylaxis before 1 year of age (30% before 6 months of age) and without concomitant FVIII (16\/20; 80%). After the loading dose, 13\/28 centres administered the recommended dosing, while the others adjusted the interval of injections to give whole vials. In inhibitor patients, the use of emicizumab during ITI was common, with low\u2010dose ITI being the preferred protoco<jats:bold>l<\/jats:bold>.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Most centres choose to initiate prophylaxis with emicizumab before 12 months of age and without concomitant FVIII. In inhibitor patients, ITI is mostly given in addition to emicizumab, but there was no common practice on how to proceed after successful ITI.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14847","type":"journal-article","created":{"date-parts":[[2023,8,30]],"date-time":"2023-08-30T16:36:12Z","timestamp":1693413372000},"page":"1291-1298","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":17,"title":["Dilemmas on emicizumab in children with haemophilia A: A survey of strategies from PedNet centres"],"prefix":"10.1111","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7854-0371","authenticated-orcid":false,"given":"Susanna","family":"Ranta","sequence":"first","affiliation":[{"name":"Childhood Cancer Research Unit Department of Women's and Children's Health Karolinska Institutet Sweden and Astrid Lindgren Children's Hospital Karolinska University Hospital  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jayashree","family":"Motwani","sequence":"additional","affiliation":[{"name":"Birmingham Children's Hospital  Birmingham UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan","family":"Blatny","sequence":"additional","affiliation":[{"name":"Department of Paediatric Haematology and Biochemistry University Hospital and Masaryk University  Brno Czech Republic"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martina","family":"B\u00fchrlen","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Klinikum Bremen Mitte  Bremen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5350-1763","authenticated-orcid":false,"given":"Manuel","family":"Carcao","sequence":"additional","affiliation":[{"name":"Haemophilia Clinic and Haemostasis Program Division of Haematology\/Oncology Department of Paediatrics Hospital for Sick Children University of Toronto  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Herv\u00e9","family":"Chambost","sequence":"additional","affiliation":[{"name":"AP\u2010HM Department of Pediatric Hematology Oncology Children Hospital La Timone &amp; Aix Marseille University INSERM INRA C2VN  Marseille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmen","family":"Escuriola","sequence":"additional","affiliation":[{"name":"Haemophilie\u2010Zentrum Rhein Main HZRM M\u00f6rfelden\u2010Walldorf  Germany"}],"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 Hematology Thrombosis and Hemostasis Van Creveldkliniek University Medical Center Utrecht University  Utrecht the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mutlu","family":"Kartal\u2010Kaess","sequence":"additional","affiliation":[{"name":"Division of Pediatric Hematology &amp; Oncology Department of Pediatrics Inselspital University Hospital Bern  Bern Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marloes","family":"de Kovel","sequence":"additional","affiliation":[{"name":"PedNet Haemophilia Research Foundation  Baarn The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1279-2557","authenticated-orcid":false,"given":"Gili","family":"Kenet","sequence":"additional","affiliation":[{"name":"The National Hemophilia Center&amp; Institute of Thrombosis &amp; Hemostasis Sheba Medical Center Tel Hashomer Israel and The Amalia Biron Research Institute of Thrombosis &amp; Hemostasis Tel Aviv University  Tel Aviv Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christoph","family":"Male","sequence":"additional","affiliation":[{"name":"Department of Paediatrics Medical University of Vienna  Vienna Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0145-4736","authenticated-orcid":false,"given":"Beatrice","family":"Nolan","sequence":"additional","affiliation":[{"name":"Children's Coagulation Centre Children's Health Ireland at Crumlin  Dublin Ireland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4843-7805","authenticated-orcid":false,"given":"Roseline","family":"d'Oiron","sequence":"additional","affiliation":[{"name":"Centre de R\u00e9f\u00e9rence de l'H\u00e9mophilie et des Maladies H\u00e9morragiques Constitutionnelles rares H\u00f4pital Bic\u00eatre AP\u2010HP et INSERM H\u00e9mostase inflammation thrombose HITH U1176 Universit\u00e9 Paris\u2010Saclay  Le Kremlin\u2010Bic\u00eatre France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6434-6244","authenticated-orcid":false,"given":"Martin","family":"Olivieri","sequence":"additional","affiliation":[{"name":"Pediatric thrombosis and Hemostasis Unit Pediatric Hemophilia Centre Dr. von Hauner Children\u00b4s Hospital LMU Munich  Munich Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ester","family":"Zapotocka","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology and Oncology University Hospital Motol Prague and 2nd Faculty of Medicine Charles University  Prague Czech Republic"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6058-8350","authenticated-orcid":false,"given":"Nadine G.","family":"Andersson","sequence":"additional","affiliation":[{"name":"Center for Thrombosis and Hemostasis Department of Clinical Sciences and Pediatrics Sk\u00e5ne 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