{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,16]],"date-time":"2026-04-16T22:58:15Z","timestamp":1776380295478,"version":"3.51.2"},"reference-count":39,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,12,31]],"date-time":"2024-12-31T00:00:00Z","timestamp":1735603200000},"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":[[2025,1]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Subcutaneous emicizumab, a factor VIII (FVIII)\u2010mimicking bispecific monoclonal antibody, can effectively prevent bleeds in haemophilia A (HA) patients with\/without inhibitors; however, its standard\u2010dose regimens are financially burdensome. Low\u2010dose emicizumab prophylaxis may alternatively be applied to noninhibitor HA patients in resource\u2010limited settings.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>During 2023, Thai patients with noninhibitor severe HA or moderate HA with severe bleeding phenotype (historical annualized bleeding rate [ABR] &gt;5 bleeds\/year before regular FVIII prophylaxis) who received low\u2010\/intermediate\u2010dose FVIII secondary prophylaxis \u22658 months were enrolled. After the 4\u2010day washout period, low\u2010dose emicizumab prophylaxis (2.0\u20132.5\u00a0mg\/kg every fortnight for two loading doses, then every 4 weeks) was implemented for 8 months. Pre\u2010\/post\u2010emicizumab ABR, annualized joint bleeding rates (AJBR), haemophilia joint health scores (HJHS) and haemophilia\u2010specific quality\u2010of\u2010life (QoL) scores were analysed. Emicizumab plasma levels on modified one\u2010stage FVIII assays were also monitored.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In 15 subjects, ABR (median of differences, \u22122 bleeds\/year; interquartile range, \u22123 to 0; <jats:italic>p<\/jats:italic> = 0.002), but not AJBR (<jats:italic>p<\/jats:italic> = 0.07), were reduced after switching to low\u2010dose emicizumab prophylaxis, although the pre\u2010dose emicizumab plasma levels at the steady state, achieved since week 12, were modest (median monthly level, 8.4\u00a0\u00b5g\/mL; interquartile range, 4.3\u201310.4). Concurrently, HJHS (<jats:italic>p<\/jats:italic> = 0.008) and QoL score (<jats:italic>p\u00a0<\/jats:italic>&lt;\u00a00.001) were decreased, and 46.7% had zero bleeds while receiving low\u2010dose emicizumab.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Low\u2010dose emicizumab, compared to low\u2010\/intermediate\u2010dose FVIII secondary prophylaxis, meaningfully improves bleeding prevention, joint health and QoL in patients with noninhibitor severe HA or moderate HA with severe bleeding phenotype. This regimen potentially helps address previously unmet needs in HA care among low\u2010to\u2010middle\u2010income countries.<\/jats:p><\/jats:sec><jats:sec><jats:title>Trial Registration<\/jats:title><jats:p>ClinicalTrials.gov identifier NCT06155955.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.15146","type":"journal-article","created":{"date-parts":[[2024,12,31]],"date-time":"2024-12-31T08:09:31Z","timestamp":1735632571000},"page":"122-131","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Low\u2010Dose Emicizumab Versus Low\u2010\/Intermediate\u2010Dose Factor VIII Secondary Prophylaxis for Noninhibitor Haemophilia A Patients With Severe Bleeding Phenotype"],"prefix":"10.1111","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0009-0007-7752-2244","authenticated-orcid":false,"given":"Nuchanun","family":"Kessakorn","sequence":"first","affiliation":[{"name":"Division of Pediatric Hematology and Oncology Department of Pediatrics Faculty of Medicine Chulalongkorn University and King Chulalongkorn Memorial Hospital  Bangkok Thailand"},{"name":"Integrative and Innovative Hematology\/Oncology Research Unit, Faculty of Medicine Chulalongkorn University and King Chulalongkorn Memorial Hospital  Bangkok Thailand"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Itsaraet","family":"Gosriwatana","sequence":"additional","affiliation":[{"name":"Chulalongkorn University International Medical Program (CU\u2010MEDi), Faculty of Medicine Chulalongkorn University  Bangkok Thailand"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nuttarak","family":"Sasipong","sequence":"additional","affiliation":[{"name":"Chulalongkorn University International Medical Program (CU\u2010MEDi), Faculty of Medicine Chulalongkorn University  Bangkok Thailand"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1951-4365","authenticated-orcid":false,"given":"Chonlatis","family":"Srichumpuang","sequence":"additional","affiliation":[{"name":"Division of Pediatric Hematology and Oncology Department of Pediatrics Faculty of Medicine Chulalongkorn University and King Chulalongkorn Memorial Hospital  Bangkok Thailand"},{"name":"Integrative and Innovative Hematology\/Oncology Research Unit, Faculty of Medicine Chulalongkorn University and King Chulalongkorn Memorial Hospital  Bangkok Thailand"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6257-0867","authenticated-orcid":false,"given":"Chatphatai","family":"Moonla","sequence":"additional","affiliation":[{"name":"Division of General Internal Medicine Department of Medicine Faculty of Medicine Chulalongkorn University and King Chulalongkorn Memorial Hospital  Bangkok Thailand"},{"name":"Center of Excellence in Translational Hematology Faculty of Medicine Chulalongkorn University and King Chulalongkorn Memorial Hospital  Bangkok Thailand"},{"name":"UNC Blood Research Center University of North Carolina at Chapel Hill  Chapel Hill North Carolina USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1097-3482","authenticated-orcid":false,"given":"Darintr","family":"Sosothikul","sequence":"additional","affiliation":[{"name":"Division of Pediatric Hematology and Oncology Department of Pediatrics Faculty of Medicine Chulalongkorn University and King Chulalongkorn Memorial Hospital  Bangkok Thailand"},{"name":"Integrative and Innovative Hematology\/Oncology Research Unit, Faculty of Medicine Chulalongkorn University and King Chulalongkorn Memorial Hospital  Bangkok 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