{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,8]],"date-time":"2026-10-08T12:39:59Z","timestamp":1791463199857,"version":"4.3.4"},"reference-count":21,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2025,10,22]],"date-time":"2025-10-22T00:00:00Z","timestamp":1761091200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp;amp; Cancer"],"published-print":{"date-parts":[[2026,1]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Hemophilia A (HA) is a rare bleeding disorder caused by coagulation factor VIII (FVIII) deficiency. Prophylactic FVIII replacement therapy is essential for preventing bleeds, but it carries a risk of inhibitor development, especially in previously untreated and minimally treated patients (PUPs and MTPs, respectively). Emicizumab, a bispecific monoclonal antibody that mimics FVIII function, offers a promising alternative for HA prophylaxis due to its subcutaneous administration and favorable safety profile.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This study evaluated the real\u2010world safety and efficacy of emicizumab prophylaxis in two international hemophilia treatment centers (HTC). The first HTC included a cohort of 22 MTPs with severe HA (FVIII &lt;1%) and fewer than five prior FVIII exposure days, while the second HTC enrolled 19 PUPs with severe HA.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The median age at emicizumab prophylaxis initiation was 5\u00a0months for the MTPs and 8\u00a0months for the PUPs. Patients were followed for a median of 27 and 29\u00a0months, respectively. The median time to first bleed was 13\u00a0months for MTPs, with a significantly longer time to first bleed noted in the PUPs cohort. Safety outcomes were favorable, with neither intracranial hemorrhage nor anti\u2010emicizumab antibodies reported. Four patients, 18% of all MTPs, developed FVIII inhibitors, all associated with high\u2010risk genetic mutations and prior bleeding events.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Our findings support the early use of emicizumab as a safe and effective prophylactic strategy in infants with severe HA. However, the observed inhibitor rate underscores the need for ongoing monitoring and research to optimize care, particularly in vulnerable populations.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/pbc.32118","type":"journal-article","created":{"date-parts":[[2025,10,23]],"date-time":"2025-10-23T04:55:28Z","timestamp":1761195328000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Emicizumab in Previously Untreated Patients and Minimally Treated Patients With Hemophilia A: A Comparative Study Between Two International Cohorts"],"prefix":"10.1002","volume":"73","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3971-0336","authenticated-orcid":false,"given":"Sarina","family":"Levy\u2010Mendelovich","sequence":"first","affiliation":[{"name":"Sheba Medical Center National Hemophilia Center and Thrombosis &amp; Hemostasis Institute  Ramat Gan Israel"},{"name":"Amalia Biron Research Institute of Thrombosis &amp; Hemostasis Gray Faculty of Medicine Tel Aviv University Tel Aviv Israel"},{"name":"Talpiot Medical Leadership Program Sheba Medical Center  Ramat Gan Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tlalit","family":"Barhod","sequence":"additional","affiliation":[{"name":"Sheba Medical Center National Hemophilia Center and Thrombosis &amp; Hemostasis Institute  Ramat Gan Israel"},{"name":"Amalia Biron Research Institute of Thrombosis &amp; Hemostasis Gray Faculty of Medicine Tel Aviv University Tel Aviv Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ivan","family":"Budnik","sequence":"additional","affiliation":[{"name":"Division of Hematology\/Oncology Department of Internal Medicine University of Iowa  Iowa City Iowa USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan","family":"Lancashire","sequence":"additional","affiliation":[{"name":"Department of Haematology Birmingham Children's Hospital  Birmingham UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesal","family":"Patel","sequence":"additional","affiliation":[{"name":"Department of Haematology Birmingham Children's Hospital  Birmingham UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8329-8409","authenticated-orcid":false,"given":"Assaf Arie","family":"Barg","sequence":"additional","affiliation":[{"name":"Sheba Medical Center National Hemophilia Center and Thrombosis &amp; Hemostasis Institute  Ramat Gan Israel"},{"name":"Amalia Biron Research Institute of Thrombosis &amp; Hemostasis Gray Faculty of Medicine Tel Aviv University Tel Aviv Israel"},{"name":"Department of Paediatric Hematology\u2010Oncology The Edmond and Lily Safra Children's Hospital, Sheba Medical Center  Ramat Gan Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Einat","family":"Avishai","sequence":"additional","affiliation":[{"name":"Sheba Medical Center National Hemophilia Center and Thrombosis &amp; Hemostasis Institute  Ramat Gan Israel"},{"name":"Amalia Biron Research Institute of Thrombosis &amp; Hemostasis Gray Faculty of Medicine Tel Aviv University Tel Aviv Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rima","family":"Dardik","sequence":"additional","affiliation":[{"name":"Sheba Medical Center National Hemophilia Center and Thrombosis &amp; Hemostasis Institute  Ramat Gan Israel"},{"name":"Amalia Biron Research Institute of Thrombosis &amp; Hemostasis Gray Faculty of Medicine Tel Aviv University Tel Aviv Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tami Brutman","family":"Barazani","sequence":"additional","affiliation":[{"name":"Sheba Medical Center National Hemophilia Center and Thrombosis &amp; Hemostasis Institute  Ramat Gan Israel"},{"name":"Amalia Biron Research Institute of Thrombosis &amp; Hemostasis Gray Faculty of Medicine Tel Aviv University Tel Aviv Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tami","family":"Livnat","sequence":"additional","affiliation":[{"name":"Sheba Medical Center National Hemophilia Center and Thrombosis &amp; Hemostasis Institute  Ramat Gan Israel"},{"name":"Amalia Biron Research Institute of Thrombosis &amp; Hemostasis Gray Faculty of Medicine Tel Aviv University Tel Aviv Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jayashree","family":"Motwani","sequence":"additional","affiliation":[{"name":"Department of Haematology Birmingham Children's Hospital  Birmingham UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gili","family":"Kenet","sequence":"additional","affiliation":[{"name":"Sheba Medical Center National Hemophilia Center and Thrombosis &amp; Hemostasis Institute  Ramat Gan Israel"},{"name":"Amalia Biron Research Institute of Thrombosis &amp; Hemostasis Gray Faculty of Medicine Tel Aviv University Tel Aviv Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,10,22]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13998"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102016\u201002\u2010683169"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.rpth.2023.100278"},{"key":"e_1_2_9_5_1","article-title":"Pharmacokinetics and Coagulation Biomarkers in Persons With Haemophilia A (PwHA) and FVIII Inhibitors Receiving Emicizumab in the Phase IIIb STASEY Study [abstract]","volume":"4","author":"Kiialainen A.","year":"2020","journal-title":"Research and Practice in Thrombosis and Haemostasis"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102017\u201008\u2010801662"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1703068"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1511769"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019001869"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.27886"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2023021832"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.30941"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.19312"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.28942"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-0039-1688687"},{"key":"e_1_2_9_16_1","unstructured":"W.Calhoon M.McInerney A.Calatzis et\u00a0al. \u201cEvaluation of a Dedicated Calibrator and Controls for Emicizumab Quantification \u201d paper presented atthe 4th Scientific Meeting of the Thrombosis and Hemostasis Societies of North America (THSNA) April 22\u201325 2018;Chicago IL."},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2023.284095"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102012\u201011\u2010469403"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2018.209619"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019000658"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1208024"},{"key":"e_1_2_9_22_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"}],"container-title":["Pediatric Blood &amp; Cancer"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pbc.32118","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,1,9]],"date-time":"2026-01-09T12:44:41Z","timestamp":1767962681000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pbc.32118"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,10,22]]},"references-count":21,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1]]}},"alternative-id":["10.1002\/pbc.32118"],"URL":"https:\/\/doi.org\/10.1002\/pbc.32118","archive":["Portico"],"relation":{},"ISSN":["1545-5009","1545-5017"],"issn-type":[{"value":"1545-5009","type":"print"},{"value":"1545-5017","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,10,22]]},"assertion":[{"value":"2025-08-01","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-10-05","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-10-22","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e32118"}}