{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,5,14]],"date-time":"2025-05-14T01:11:28Z","timestamp":1747185088380,"version":"3.40.5"},"reference-count":35,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2023,5,30]],"date-time":"2023-05-30T00:00:00Z","timestamp":1685404800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["pericles.pericles-gcp.literatumonline.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>The timing of prophylaxis and <jats:italic>F8<\/jats:italic> genotype can impact treatment outcomes in adults with severe haemophilia A (HA).<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To investigate how <jats:italic>F8<\/jats:italic> genotype, timing, and type of prophylaxis influence arthropathy, bleeding rates, factor consumption and health\u2010related quality of life (HRQoL).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Thirty\u2010eight patients with severe HA were enrolled. Bleeding events were recorded retrospectively during median 12.5 months. <jats:italic>F8<\/jats:italic> gene variants were classified as null or non\u2010null. Joint health and HRQoL were assessed with HJHS and EQ\u20105D\u20105L, respectively.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The median age at prophylaxis start was 1.25 years in the primary prophylaxis group (<jats:italic>N<\/jats:italic>\u00a0=\u00a015, median age 26 years) and 31.5 years in the secondary group (<jats:italic>N<\/jats:italic>\u00a0=\u00a022, 45 years), respectively. There were significant differences in the medians of HJHS (4 vs. 20, <jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.001), EQ\u20105D\u20105L index (0.9647 vs. 0.904, <jats:italic>p<\/jats:italic>\u00a0=\u00a0.022), EQ VAS (87 vs. 75, <jats:italic>p<\/jats:italic>\u00a0=\u00a0.01) and FVIII consumption (3883 vs. 2737 IU\/kg\/year, <jats:italic>p<\/jats:italic>\u00a0=\u00a0.02), between the primary and secondary groups, respectively. Median annualized bleeding rate (ABR) was 0 for both groups. Twenty\u2010five null and thirteen non\u2010null <jats:italic>F8<\/jats:italic> gene variants were identified. In the secondary prophylaxis group, lower median FVIII consumption (1926 vs. 3370 IU\/kg\/year) was shown for non\u2010null compared to null variants, respectively,\u00a0with similar ABR and HJHS.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Delayed prophylaxis start with intermediate dose intensity prevents bleeds but at a cost of more arthropathy and reduced HRQoL, compared to higher intensity primary prophylaxis. Non\u2010null <jats:italic>F8<\/jats:italic> genotype may allow lower factor consumption with similar HJHS and bleeding rates, compared to null genotype.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14806","type":"journal-article","created":{"date-parts":[[2023,5,30]],"date-time":"2023-05-30T17:11:25Z","timestamp":1685466685000},"page":"1032-1038","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Impact of timing of prophylaxis commencement, F8 genotype and age on factor consumption and health\u2010related quality of life in patients with severe haemophilia A"],"prefix":"10.1111","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9371-9396","authenticated-orcid":false,"given":"Alexandros","family":"Arvanitakis","sequence":"first","affiliation":[{"name":"Department of Translational Medicine Lund University  Malm\u00f6 Sweden"},{"name":"Department of Hematology Oncology and Radiation Physics Sk\u00e5ne 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