{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,3]],"date-time":"2026-05-03T07:24:44Z","timestamp":1777793084705,"version":"3.51.4"},"reference-count":31,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2023,10,11]],"date-time":"2023-10-11T00:00:00Z","timestamp":1696982400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Emicizumab mimicking the cofactor function of activated factor VIII (FVIII) restores haemostasis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This nationwide observational study aimed to retrospectively investigate efficacy, safety, and cost in 1\u00a0year before and up to 3\u00a0years after emicizumab prophylaxis for haemophilia A (HA) patients with FVIII inhibitors.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results and discussion<\/jats:title><jats:p>A total of 39 severe HA patients with a median age of 23.0\u00a0years were enrolled. The median historical peak FVIII inhibitor titre was 174.2\u00a0BU\/mL with an interquartile range of 56.5\u2013578.8\u00a0BU\/mL. The median annualized bleeding rate reduced from 24 to 0 events in the first year after emicizumab prophylaxis (<jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.01) and sustained in the second and third years. The median annualized joint bleeding rate reduced to 0 and maintained up to 3\u00a0years (<jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.01). Twenty\u2010seven patients (69.2%) had target joints before emicizumab prophylaxis and only seven patients (17.9%) of them had target joints after prophylaxis. Medical costs, including cost of haemostatic therapy, frequency of outpatient department visits, emergency room visits and hospital admission, were significantly reduced after emicizumab prophylaxis (<jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.01). FVIII inhibitor titre decreased after emicizumab prophylaxis. Overall, three (7.7%) patients experienced 202 grade 1 drug\u2010related adverse events after emicizumab prophylaxis. No serious adverse events were reported during emicizumab prophylaxis period. The adherence to emicizumab prophylaxis was 100% up to 3\u00a0years.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>HA patients with FVIII inhibitors treated with emicizumab prophylaxis resulted in a significant reduction in treated bleeds and associated costs. No new safety events were observed.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14880","type":"journal-article","created":{"date-parts":[[2023,10,12]],"date-time":"2023-10-12T05:37:51Z","timestamp":1697089071000},"page":"1499-1508","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Efficacy, safety and cost of emicizumab prophylaxis in haemophilia A patients with inhibitors: A nationwide observational study in Taiwan"],"prefix":"10.1111","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7789-2148","authenticated-orcid":false,"given":"Ming\u2010Ching","family":"Shen","sequence":"first","affiliation":[{"name":"Department of Internal Medicine Changhua Christian Hospital  Changhua Taiwan"},{"name":"Division of Hematology, Department of Internal Medicine National Taiwan University Hospital  Taipei 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