{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,20]],"date-time":"2026-01-20T17:08:44Z","timestamp":1768928924204,"version":"3.49.0"},"reference-count":36,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2024,4,5]],"date-time":"2024-04-05T00:00:00Z","timestamp":1712275200000},"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":[[2024,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Aim<\/jats:title><jats:p>To characterise non\u2010severe haemophilia A (HA) patients enrolled on the Australian Bleeding Disorders Registry (ABDR) treated through a state\u2010wide Haemophilia Treatment Centre (HTC) with respect to their mutational profile, inhibitor risk and health\u2010care burden.<\/jats:p><\/jats:sec><jats:sec><jats:title>Method<\/jats:title><jats:p>We conducted a single\u2010centre observational study of all non\u2010severe HA patients treated at the Alfred Health HTC registered on the ABDR as of the 26th\u00a0July 2023. Data were extracted from the ABDR and electronic medical record (EMR) regarding demographics, severity, genetic testing, treatment, inhibitors, bleeding events and procedures. Inhibitor risk was calculated as a function of exposure days (EDs) of FVIII replacement.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>There were 289 non\u2010severe HA patients treated at the Alfred HTC registered on the ABDR as of July 2023, all of whom were adult patients aged\u00a0&gt;\u00a018 years old. Genotyping had been performed in 228\/289 (78.9%). Of the inhibitor analysis population, 14\/193 (7.3%) had an inhibitor. The cumulative incidence of inhibitor development at 75 EDs was 31% (95% CI 13%\u201346%). The median cost of bypassing agents per inhibitor patient was $57,087.50\/year.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>These results demonstrate a relatively high inhibitor prevalence and incidence risk in non\u2010severe HA compared to previously published work, although this may partly reflect a smaller population size. High rates of genotyping have allowed representative mutational characterisation. The burden of care imposed by non\u2010severe HA in terms of bleeding events, procedures and bypassing agent cost is larger than expected, particularly within the inhibitor population.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.15007","type":"journal-article","created":{"date-parts":[[2024,4,5]],"date-time":"2024-04-05T13:38:43Z","timestamp":1712324323000},"page":"638-647","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Mutational landscape, inhibitor development, and health\u2010care burden in non\u2010severe haemophilia A: A single\u2010centre Australian experience"],"prefix":"10.1111","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5539-0673","authenticated-orcid":false,"given":"Radha","family":"Ramanan","sequence":"first","affiliation":[{"name":"Australian Centre for Blood Diseases Monash University  Melbourne Victoria Australia"},{"name":"Ronald Sawers Haemophilia Treatment Centre Department of Haematology Alfred Hospital  Melbourne Victoria Australia"},{"name":"Australian Haemophilia Centre Directors\u2019 Organisation  Melbourne Victoria Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Natalie","family":"Evans","sequence":"additional","affiliation":[{"name":"Ronald Sawers Haemophilia Treatment Centre Department of Haematology Alfred Hospital  Melbourne Victoria Australia"},{"name":"Australian Haemophilia Centre Directors\u2019 Organisation  Melbourne Victoria Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zane","family":"Kaplan","sequence":"additional","affiliation":[{"name":"Ronald Sawers Haemophilia Treatment Centre Department of Haematology Alfred Hospital  Melbourne Victoria Australia"},{"name":"Department of Clinical Haematology Monash Health  Melbourne Victoria Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"James D.","family":"McFadyen","sequence":"additional","affiliation":[{"name":"Australian Centre for Blood Diseases Monash University  Melbourne Victoria Australia"},{"name":"Ronald Sawers Haemophilia Treatment Centre Department of Haematology Alfred Hospital  Melbourne Victoria Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Huyen A.","family":"Tran","sequence":"additional","affiliation":[{"name":"Australian Centre for Blood Diseases Monash University  Melbourne Victoria Australia"},{"name":"Ronald Sawers Haemophilia Treatment Centre Department of Haematology Alfred Hospital  Melbourne Victoria Australia"},{"name":"Australian Haemophilia Centre Directors\u2019 Organisation  Melbourne Victoria Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2024,4,5]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199401063300108"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12672"},{"key":"e_1_2_13_4_1","unstructured":"National Blood Authority.Australian Bleeding Disorders Registry Annual Report 2020\u20132021 [Internet]. 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