{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T04:54:59Z","timestamp":1790571299365,"version":"4.1.0"},"reference-count":25,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2025,12,12]],"date-time":"2025-12-12T00:00:00Z","timestamp":1765497600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2025,12,12]],"date-time":"2025-12-12T00:00:00Z","timestamp":1765497600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2026,1]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>The bispecific monoclonal antibody emicizumab was approved for prophylactic treatment of congenital haemophilia A (HA) in Japan in 2018.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>To monitor long\u2010term safety and effectiveness of emicizumab, including appropriate concomitant use of bypassing agents (BPAs), in Japanese patients with congenital HA with inhibitors who initiated emicizumab within 1 year of availability.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This all\u2010case post\u2010marketing surveillance (PMS) study was conducted between May 2018 and January 2023, in patients of all ages and HA severities. Patients were registered retrospectively after emicizumab initiation or prospectively at the time of initiation. Adverse events (AEs) and adverse drug reactions (ADRs), including thromboembolic events (TEs) and thrombotic microangiopathy (TMA), and their association with BPA use, were examined. FVIII inhibitor levels and annualised bleeding rates (ABRs) were evaluated.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>In total, 134 patients were included in the analysis. Mean (standard deviation) duration of emicizumab treatment was 145.5 (34.8) weeks. Overall, 112 AEs occurred in 47 patients (35.1%) and 22 ADRs occurred in 12 patients (9.0%); 8 ADRs (36.4%) were serious. No TEs\/TMAs associated with concomitant use of BPAs occurred. FVIII inhibitor levels remained stable or decreased for 34 patients (55.7%). Sixty\u2010eight patients (50.7%) received BPAs; none received activated prothrombin complex concentrate. Mean (95% confidence interval) model\u2010based all bleed ABR and treated bleed ABR were 1.4 (0.9\u20132.3) and 1.3 (0.8\u20132.1), respectively.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>This study comprises 3 years\u2019 PMS for emicizumab. No TEs\/TMA associated with BPAs occurred. Results further support the safety and effectiveness of emicizumab in Japanese patients with congenital HA with inhibitors.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.70162","type":"journal-article","created":{"date-parts":[[2025,12,13]],"date-time":"2025-12-13T05:27:33Z","timestamp":1765603653000},"page":"135-142","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Post\u2011Marketing Surveillance of the Safety and Effectiveness of Emicizumab in Japanese Patients With Congenital Haemophilia A With Inhibitors"],"prefix":"10.1111","volume":"32","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5922-7061","authenticated-orcid":false,"given":"Midori","family":"Shima","sequence":"first","affiliation":[{"name":"Thrombosis and Hemostasis Research Center Nara Medical University  Kashihara Japan"},{"name":"Department of Pediatrics Nara Medical University  Kashihara Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katsuyuki","family":"Fukutake","sequence":"additional","affiliation":[{"name":"Department of Blood Coagulation Ogikubo Hospital  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masanori","family":"Matsumoto","sequence":"additional","affiliation":[{"name":"Department of Blood Transfusion Medicine Nara Medical University  Kashihara Japan"},{"name":"Department of Hematology Nara Medical University  Kashihara Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshihiko","family":"Sugimura","sequence":"additional","affiliation":[{"name":"Drug Safety Division Chugai Pharmaceutical Co., Ltd  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiaki","family":"Sugita","sequence":"additional","affiliation":[{"name":"Drug Safety Division Chugai Pharmaceutical Co., Ltd  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lyu","family":"Ji","sequence":"additional","affiliation":[{"name":"Drug Safety Division Chugai Pharmaceutical Co., Ltd  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mika","family":"Kawano","sequence":"additional","affiliation":[{"name":"Medical Affairs Division Chugai Pharmaceutical Co., Ltd  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tadashi","family":"Matsushita","sequence":"additional","affiliation":[{"name":"Department of Transfusion Medicine Nagoya University Hospital  Nagoya Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,12,12]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(15)01123-X"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_12_4_1","unstructured":"The Haemophilia Society.Bleeding Disorders\u2014Inhibitors. (2024) Accessed September 2025 https:\/\/haemophilia.org.uk\/bleeding\u2010disorders\/inhibitors."},{"key":"e_1_2_12_5_1","unstructured":"F. Hoffmann\u2010La Roche Ltd.Emicizumab Summary of Product Characteristics. (2024). Accessed September 2025 https:\/\/www.ema.europa.eu\/en\/documents\/product\u2010information\/hemlibra\u2010epar\u2010product\u2010information_en.pdf."},{"key":"e_1_2_12_6_1","unstructured":"Genentech Inc.HEMLIBRA\u00ae(emicizumab\u2010kxwh) Injection for Subcutaneous Use Prescribing Information. (2024) Accessed September 2025 https:\/\/www.gene.com\/download\/pdf\/hemlibra_prescribing.pdf."},{"key":"e_1_2_12_7_1","unstructured":"Pharmaceutical and Medical Devices Agency.Hemlibra Prescribing Information. Accessed September 2025 https:\/\/www.pmda.go.jp\/PmdaSearch\/iyakuDetail\/GeneralList\/6343451."},{"issue":"5","key":"e_1_2_12_8_1","first-page":"475","article-title":"[Emicizumab, a Bispecific Antibody Mimicking Factor VIII: A Novel Alternative Therapy for Hemophilia A With Inhibitors] Rinsho Ketsueki","volume":"60","author":"Nogami K.","year":"2019","journal-title":"Japanese Journal of Clinical Hematology"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1703068"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019001869"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1803550"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(19)30054-7"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2022.992267"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2022-157531"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020009217"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.rpth.2024.102364"},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12837"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14205"},{"key":"e_1_2_12_19_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13848"},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-023-36891-x"},{"key":"e_1_2_12_21_1","first-page":"98","article-title":"Emicizumab Prophylaxis in People With Haemophilia A: Summary of 10 Years of Safety Data on Thromboembolic Events and Thrombotic Microangiopathy","volume":"30","author":"Sarouei K.","year":"2024","journal-title":"Haemophilia"},{"key":"e_1_2_12_22_1","first-page":"3668","article-title":"Use of Bypassing Agents Prior to and Post Bypassing Agent Dosing Guidance During Emicizumab Prophylaxis: Analyses From the HAVEN 1 Study","volume":"130","author":"Callaghan M.","year":"2017","journal-title":"Blood"},{"key":"e_1_2_12_23_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14762"},{"key":"e_1_2_12_24_1","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2021-059667"},{"key":"e_1_2_12_25_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2022-157249"},{"key":"e_1_2_12_26_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.70070"}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.70162","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/hae.70162","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.70162","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T03:55:25Z","timestamp":1790567725000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.70162"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,12,12]]},"references-count":25,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1]]}},"alternative-id":["10.1111\/hae.70162"],"URL":"https:\/\/doi.org\/10.1111\/hae.70162","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"value":"1351-8216","type":"print"},{"value":"1365-2516","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,12,12]]},"assertion":[{"value":"2025-03-14","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-10-24","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-12-12","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}